Message from Founder. December Special. End of Year Giving. Research

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1 December 2014 Special Message from the Founder Message from Founder End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital BioMarin and Prosensa Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare- Disease Portfolio Media Statement Regarding BioMarin and Prosensa Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients Debra and Hawken Miller This year has been an eventful one for the Duchenne community. Every year we hope and pray this will be the year that we can say there is a cure for Duchenne. Each year we get closer to an approved treatment. and development takes time and has its ups and downs as you can see from this year s research news. Here are some research highlights from funded projects in 2014: Prosensa regained the rights of drisapersen from GSK and retains rights to all other programs Sarepta Therapeutics announced plans for eteplirsen to submit NDA by the end of 2014 Akashi Therapeutics received fast track designation for HT-100 from FDA Ventures funds first social impact investment with Prosena announced $7 million collaboration with Prosensa to accelerate access to treatments for Duchenne Dr. Kevin Flanigan from Nationwide Children s Hospital published report with results of a study funded by in Nature Medicine PTC Therapeutics received conditional approval in the European Union for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy Prosensa commenced re-dosing of drisapersen in North American in patients with Duchenne muscular dystrophy Sarepta initiated Phase III clinical study for eteplirsen Sarepta announced plans to submit an NDA by mid-year 2015, pending any additional requests from further discussions with the FDA Prosensa began NDA submission to the FDA for exon-skipping drug drisapersen to treat Duchenne muscular dystrophy Prosensa extended the re-dosing of drisapersen in Europe in patients with Duchenne muscular dystrophy Sarepta began dosing in confirmatory study of eteplirsen in ambulant

2 PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into Clinical Stage Program Targeting News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports patients with Duchenne muscular dystrophy Sarepta initiated dosing in a clinical study of eteplirsen in non-ambulant patients with Duchenne muscular dystrophy BioMarin acquired Prosensa and added Duchenne muscular dystrophy products to its rare disease portfolio As the year comes to a close, Paul and I would like to thank all supporters for your support over the years. We will continue to fund research until we find a cure for all boys with Duchenne. The good news for boys with Duchenne is that there are potential treatments on the horizon. The bad news is this generation of boys will stop walking or die before they see the benefit of these drugs unless they can receive them soon. These boys cannot wait. If you have donated to our year end campaign, we thank you for your generosity. Your contribution gives hope to the 300,000 boys worldwide with Duchenne and their families. If you haven t donated yet we could still use your support. Please go to: Or mail a check to:, 1400 Quail St. #110, Newport Beach, CA Donations in stock are also accepted. Best wishes to you and your family for a joyous holiday season and a happy, healthy With gratitude, Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Debra Miller Founder and CEO Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

3 December 2014 Special Message from the Founder End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

4 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

5 December 2014 Special Message from the Founder End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

6 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

7 December 2014 Special Message from the Founder End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital BioMarin and Prosensa Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare- Disease Portfolio Media Statement Regarding BioMarin and Prosensa Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients Kevin M. Flanigan, MD (far right), a national nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, announced today that they are sponsoring a $710,000 research project with Kevin Flanigan, M.D., principal investigator at the Center for Gene Therapy at Nationwide Children s Hospital in Columbus, Ohio, that focuses on the development of a novel therapy for duplication mutations for Duchenne muscular dystrophy. The project will fund the production of the drug substance (an adeno-associated virus AAV9.U7) that will be used in the IND-enabling toxicology/biodistribution studies. Dr. Flanigan s research focuses on exon skipping for duplication mutations. Duchenne is a progressive muscle-wasting disease that is estimated to affect 1 in 3,500 boys. Muscle degeneration is due to an absence of a protein in the muscle called dystrophin. Dystrophin is a large gene with 79 exons. The exons get spliced together to form a final blueprint. In Duchenne patients, the blueprint cannot be read from start to finish so the body can t make the protein. Exon skipping alters the splicing pattern in a gene containing a Duchenne mutation in order to restore the reading frame. This would give a boy with Duchenne a shorter, but still functional dystrophin protein. The goal is to take out the duplicated exon and make it normal, which is called a wild type dystrophin, to restore a normal blueprint. Dr. Flanigan s research focuses on the clinical development of an U7snRNA vector for exon 2 skipping; therapy for exon 2 duplications and the five prime mutations on the Duchenne muscular dystrophy gene. Dr. Flanigan s lab has identified a novel element in the dystrophin gene that may allow the rescue of mutations that are way up in front of the gene the 5 region of the gene. This element, called the Internal Ribosome Entry Site (IRES), allows translation of a protein from a starting site within exon 6. It gives a large therapeutic window for the idea of skipping exon 2. This is because skipping of only one copy of exon 2 would give a normal gene message, and complete skipping of exon 2 would activate the IRES and result in the translation of a highly functional shorter version of the protein. These results not only provide a great deal of confidence in the usefulness of this vector for patients with duplications of exon 2, but also provide a potential

8 PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into Clinical Stage Program Targeting News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media treatment for any patients with mutations within the first 5 exons of the gene. Results of Dr. Flanigan s study were recently published in the journal Nature Medicine, and was supported by funding from. In 2011, provided the funding for Dr. Flanigan to create a new Duchenne mouse model with a duplication mutation of exon 2, providing the first animal model in which to directly experiment with exon skipping for duplication mutations. These results provide Dr. Flanigan s lab with a pathway forward to treat patients with a duplication of exon 2, and also patients with mutations within the first five exons of the gene. recognized early on the importance of our research focused on rare and duplication mutations and we wouldn t be here today without their support, said Dr. Kevin Flanigan. supported the development of the mouse model and the personnel to work on this research. plays a vital role in funding early phase research projects to help accelerate their development. This new research project continues s support of Dr. Flanigan s research and is part of s comprehensive strategy of funding Duchenne research that could potentially lead to treatments and a cure for Duchenne. has funded all types of Duchenne research including exon skipping, rare mutations, stop codon, anti-fibrotic therapies, utrophin upregulation and anti-inflammatory. Boys with Duchenne are usually diagnosed by age 5, in a wheelchair by 12 and most don t survive their mid-20s. There is currently no cure for Duchenne. Dr. Flanigan s research expands our understanding of the genetic underpinnings of Duchenne and will help uncover new paths toward a cure, said Debra Miller, CEO and founder of. We want a cure for all affected by the disease, and that is why we have supported all duplication and rare mutations. is pleased to see Dr. Flanigan s work progress and is proud to continue to support his important work. We encourage Duchenne families and other donors to help us fund this important research. Learn more information about the Dr. Flanigan project at and donate to this project. Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

9 December 2014 Special Message from the Founder End of Year Giving Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into Acquisition of Prosensa provides near-term opportunity to commercialize, if approved, its exon-skipping drug candidate, drisapersen, for Duchenne muscular dystrophy (DMD) Drisapersen is currently under a rolling review as part of a New Drug Application process and has Orphan, Fast Track and Breakthrough Therapy designation by the FDA Drisapersen, a potential first-to-market and best-in-class product for treating a large population of patients with a rare, fatal genetic disease represents up to 10,000 DMD patients Follow-on products leveraging Prosensa's same technology platform in the pipeline target an additional 35,000 DMD patients in BioMarin's commercial territories BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) and Prosensa Holding N.V. (Nasdaq:RNA) announced on November 24 that they have entered into a definitive agreement in which BioMarin will offer to purchase all of the outstanding ordinary shares of Prosensa for $17.75 per share, for a total up front consideration of approximately $680 million. In addition, two approximately $80 million contingent milestones are payable for the approval of drisapersen in the U.S. no later than May 15, 2016 and Europe no later than February 15, 2017, respectively. "BioMarin is dedicated to the rare disease community, and the acquisition of Prosensa fits strategically with our mission of delivering therapies that address serious unmet medical needs," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "We are committed to working closely with regulatory authorities worldwide in bringing a potentially breakthrough therapy to patients with this devastating condition." Mr. Bienaimé continued, "We will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible. Further, if we are successful in advancing drisapersen to early regulatory approvals, we believe this transaction would be accretive to operating and GAAP profitability in 2017." Under the terms of the definitive agreement, BioMarin will offer to acquire all of Prosensa's issued and outstanding ordinary shares and all ordinary share equivalents in an all cash transaction for $17.75 per share for an upfront purchase price of approximately $680 million. Prosensa shareholders may also receive two regulatory milestone payments of approximately $80 million for receiving approval in the U.S. no later than May 15, 2016 and in Europe no later than February 15, 2017, respectively. In addition, within 5 business days of signing the

10 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif. purchase agreement BioMarin will purchase from Prosensa a $50 million convertible note. If the transaction fails to close for any reason, the note will automatically convert into 4,395,914 shares of Prosensa's stock. The transaction is expected to be accounted for as a business combination. BioMarin will maintain operations at Prosensa's headquarters, based in Leiden, The Netherlands and integrate Prosensa personnel from that office. The acquisition will provide BioMarin with worldwide rights to multiple orphan-drug candidates, including drisapersen, which is currently under rolling review as part of a New Drug Application (NDA) with the Food and Drug Administration. Prosensa's pipeline is comprised of several potential products that leverage their proprietary RNA-modulating technology platform for the treatment of various genotypes of Duchenne muscular dystrophy and other genetic disorders. Hans Schikan, Chief Executive Officer of Prosensa added, "BioMarin has established itself as a leader in rare diseases, characterized by strong management, thorough execution, and a resounding commitment to patients in developing and commercializing treatments where there is a high unmet medical need. This transaction will enhance Prosensa's mission by bringing innovative therapies to patients across the world as quickly and efficiently as possible. The deal also creates shareholder value by positioning Prosensa's strong portfolio of orphan drug candidates for future success with a prominent rare disease company that has the experience and dedication to bring drisapersen and our follow-on compounds to the hands of patients who desperately need them." BioMarin will effect the transaction primarily through a tender offer for all of the issued and outstanding Prosensa ordinary shares (the "Offer") and expect to close in the first quarter of The commencement of the Offer will be subject to having obtained workers council advice, and the consummation of the Offer is subject to the satisfaction of customary closing conditions for a transaction of this nature, including the tender of at least 80% of the issued and outstanding Prosensa ordinary shares and the receipt of regulatory clearance. Following completion of the Offer, the Supervisory Board of Prosensa will consist of five individuals designated by BioMarin and two individuals who currently serve on the Supervisory Board of Prosensa, who will act as independent directors. The two independent directors will, in accordance with Dutch practice, act as independent supervisory directors to protect the interest of any minority shareholders until BioMarin utilizes certain available reorganization structures available under Dutch law to acquire full ownership of Prosensa's outstanding shares and/or its business. An Extraordinary General Meeting will be convened in connection with the Offer and to adopt, among other things, certain resolutions relating to the reorganization of Prosensa.

11 December 2014 Special Message from the Founder End of Year Giving Media Statement Regarding Announcement to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne is encouraged about BioMarin Pharmaceutical s acquisition of Prosensa and their commitment to continue the development of drugs for Duchenne muscular dystrophy. BioMarin has a deep understanding of the orphan rare disease space, experience working with regulatory authorities worldwide and a successful record of getting drugs approved. supports all research efforts that further the development of treatment options for boys with Duchenne. has played a critical role in helping to fund Prosensa for more than 10 years that has helped bring drisapersen to its current stage of development. Drisapersen is currently under a rolling review as part of the New Drug Application process. has also funded Prosensa s other exon skipping compounds that are under development. has been a dedicated supporter of Prosensa since our company s inception, said Hans Schikan, Chief Executive Officer of Prosensa. s funding came at critical times during our drug development process and their longtime commitment has been of enormous value to the company. The transaction with BioMarin will enhance our mission by bringing innovative therapies to patients across the world as quickly as possible. Our commitment to finding treatments for Duchenne is unwavering, said Debra Miller, Founder and CEO of. We have been there every step of the way for Prosensa and are encouraged that BioMarin will continue this effort that will hopefully lead to approval of drisapersen and a pipeline of other follow on exons. Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

12 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

13 December 2014 Special Message from the Founder End of Year Giving Offers Continued Support to BioMarin for Duchenne to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, will continue to watch the performance of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) during its acquisition of Prosensa, a biotechnology company has supported at critical times. On December 10, BioMarin hosted its first Analyst & Investor Day since the acquisition announcement November 24, and will be providing updates on the company's pipeline and commercial programs. uses venture philanthropy offering venture capital to pharmaceutical companies to help fund Duchenne research. was an early supporter of research into exon skipping, (a method used to cause cells to skip over faulty sections of genetic code, leading to a shorter but still functional protein), and has played a critical role in helping to fund Prosensa s exon skipping drug drisapersen. is invigorated by BioMarin s commitment to continue the development of such drugs. looks forward to working with BioMarin, and we support their efforts to further the development of treatment options for boys with Duchenne, said Debra Miller, Founder and CEO of. We continue to use our venture philanthropy model to fund research to help accelerate access to drugs and expedite the approval of innovative therapies to treat this generation of Duchenne boys. has supported Prosensa since the company s inception and funded Prosensa at critical times in the development of new drugs. Drisapersen is currently under a rolling review as part of the New Drug Application process. recently funded other exon skipping compounds that are under development. We are dedicated to funding research to find a cure for Duchenne, said Miller. For the Duchenne community, the science can t move fast enough. With BioMarin s experience in the orphan rare disease space we are hopeful that there will soon be an approved treatment for this devastating disease. Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

14 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

15 December 2014 Special Message from the Founder End of Year Giving Pfizer Initiates Phase 2 Study of PF in to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Pfizer Inc. (NYSE:PFE) announced today enrollment of the first patient in a multicenter Phase II clinical trial of the investigational compound PF in boys with Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. PF is an experimental, infused, anti-myostatin monoclonal antibody. Myostatin is a naturally occurring protein in muscles that helps control muscle growth; it is believed that blocking the activity of myostatin may have potential therapeutic application in treating muscle wasting diseases such as DMD. DMD is a devastating and debilitating disease impacting approximately 1 in 3,500 male births worldwide with no current treatment options, said Kevin Lee, Ph.D., senior vice president and chief scientific officer of Pfizer s Rare Disease Unit. We are pleased to be taking this important next step in the development of PF as an investigational therapy for DMD in the hopes of potentially bringing a much-needed therapy to individuals and families with this devastating disease. The phase 2 clinical trial will evaluate the safety, tolerability and efficacy of PF in boys aged 6 to 10 years old diagnosed with DMD regardless of genotype. Based on the proposed mechanism of action of PF , Pfizer is exploring whether there is the potential to increase muscle mass and function in boys with DMD who are weak and have lost muscle. Read more about this study in letter from Pfizer and frequently asked questions. We are delighted to have Pfizer working on developing potential treatments to help find a cure for Duchenne. We wish them much success in this trial. Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

16 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

17 December 2014 Special Message from the Founder End of Year Giving Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit (AIM: SUMM), the drug discovery and development company advancing therapies for ('DMD') and C. difficile infection ('CDI'), announces that it has received approval from the UK Medicines and Healthcare products Regulatory Agency and the Ethics Review Committee to initiate a Phase 1b modified diet trial of SMT C1100. SMT C1100 is an oral small molecule utrophin modulator in development for the potential treatment of all patients with DMD, regardless of the underlying dystrophin fault causing the disease. This new trial aims to increase the plasma levels of SMT C1100 by providing patients with specific dietary guidance intended to improve drug absorption. The trial will also evaluate the potential impact that SMT C1100 is having on enzyme markers of muscle health. Top-line data from the Phase 1b modified diet trial are expected to be reported in mid "We believe it is possible to enhance absorption of SMT C1100 through dietary means and this new patient trial is designed to test this so that we can confidently evaluate the efficacy of utrophin modulation in subsequent clinical trials," commented Glyn Edwards, Chief Executive Officer. "We believe that utrophin modulation has the opportunity to benefit all boys with DMD and we are working to ensure this molecule has the best chance to reach the market through a well-designed clinical path." Read more. Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

18 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

19 December 2014 Special Message from the Founder End of Year Giving PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on December 3 that Translarna (ataluren) is now commercially available to patients in Germany, the first country to launch in the European Union (EU), with first shipments expected to begin this week. "The launch of Translarna in Germany marks another exciting milestone for the DMD community," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "Through our reimbursed early access programs and commercial efforts, we are working hard to bring Translarna to those who may benefit from this drug. We remain committed to bringing Translarna to patients in Europe, and around the world, as quickly as possible. We know every day counts for the patients and their families." Read more. Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

20 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

21 December 2014 Special Message from the Founder End of Year Giving ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into Clinical Stage Program Targeting to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients ARMGO Pharma and Servier announced on December 4 the successful completion of preclinical efficacy and IND/CTA enabling studies with ARM210/S48168, along with a formal decision to advance the program into early clinical development initially targeting treatment for patients with (DMD), the most common and severe form of muscular dystrophy. ARMGO Pharma and Servier have collaborated since 2006 on the advancement of a novel class of small molecule drugs known as Rycals, which were initially discovered through the pioneering work of Dr. Andrew R. Marks, the Clyde and Helen Wu Professor of Molecular Cardiology (in Medicine), Chairman of the Department of Physiology and Cellular Biophysics, and Founding Director of the Clyde and Helen Wu Center for Molecular Cardiology at Columbia University Medical Center. Rycals target the Ryanodine Receptor (RyR), an intracellular calcium release channel that becomes leaky in disease states including, contributing to muscle damage and loss of function. Rycals have been shown in animal models of muscle disease to repair RyR-mediated intracellular calcium leak and thereby improve muscle specific force and exercise capacity. Further, Rycals have been shown to exert similar beneficial effects on cardiac muscle in animal models of heart disease. The proprietary drug candidate ARM210/S48168, discovered by ARMGO Pharma, was selected by the ARMGO-Servier collaboration from a library of Rycal candidates for preclinical advancement as a potential treatment for DMD and other muscle disorders. With its unique mechanism of action and oral delivery formulation, ARM210/S48168 has the potential to provide benefit across skeletal muscle, diaphragm and heart muscle in DMD patients regardless of genetic background, both as a mono-therapy as well as in conjunction with other treatments. Read more. PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

22 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

23 December 2014 Special Message from the Founder End of Year Giving Clay Matthews of the Green Bay Packers Featured in New PSA Clay Matthews - PSA to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients announced on December 18 that Clay Matthews of the Green Bay Packers is featured in a new Public Service Announcement to help raise awareness about Duchenne muscular dystrophy. The PSA is part of s ongoing efforts to raise awareness and fund research to find a cure for Duchenne, a progressive muscle-wasting disease that impacts 1 in every 3,500 boys. Boys are usually diagnosed by the age of 5, in a wheelchair by 12 and most don t survive their mid-20s. Currently, there is no cure for Duchenne. For someone who puts in a lot of hard work to build up my muscles and on the contrary to have boys and young men with Duchenne have the exact opposite taken away from them is disheartening, said Matthews. That is why I m trying to spread awareness to get the funding we need to be that much closer to having a cure. Please donate and help cure Duchenne. The 60-second PSA can be viewed online on s YouTube channel. Click to view or to donate. We are delighted that Clay Matthews continues to be a big supporter of our efforts to fund research to find a cure for Duchenne, said Debra Miller, Founder and CEO of. Clay has a big heart and we appreciate his compassion to help those who live with Duchenne. We will continue to fund research to help accelerate access to drugs until there is a treatment for all boys with Duchenne. PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

24 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

25 December 2014 Special Message from the Founder Napa in Newport Wine Auction End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital BioMarin and Prosensa Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare- Disease Portfolio Media Statement Regarding BioMarin and Prosensa Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients Napa Valley is coming to Newport Beach for the Napa in Newport Beach Wine Auction on February 28, Enjoy the finest Napa wines and farm-to-table dinner with renowned Napa vintners at each table. This wine and food inspired event benefits. More than 20 Napa Valley vintners including Vineyard 29, Barbour Wines, Broman Cellars, Cliff Lede Vineyard, Dana Estate, Darioush, Heitz Cellars, Lail Vineyards and more will showcase their finest wines. Guests will have an opportunity to taste a variety of Napa wines and talk with the vintner at their table.

26 PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into Clinical Stage Program Targeting The exclusive event will be held at the Balboa Bay Resort in Newport Beach, Calif. The evening features a wine tasting reception, silent auction, dinner and wine pairings, program and live auction lots with one-of-a-kind wine themed experiences and After Hours party. Check and Anne McMinn, owners of Vineyard 29, are honorary co-chairs of the Napa in Newport Wine Auction. The wine curators is Master Sommelier Michael Jordan. For more information or to buy tickets, News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

27 December 2014 Special Message from the Founder End of Year Giving Holiday Shopping at Amazon Smile Supports to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement When you are doing your holiday shopping, please consider using Amazon Smile and support. Amazon Smile will donate 0.5% of all qualifying purchases to. Millions of product qualify, and will be indicated on the item pages. There is no additional cost to you! Please go directly to to support. Make your purchase as usual. Amazon Smile gives you the same great benefits of Amazon, but you re supporting. Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

28 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

29 December 2014 Special Message from the Founder End of Year Giving Marching Bands Perform Run Away With Sarah Burgess to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into Singer-songwriter Sarah Burgess performed Run Away with several marching bands this fall. Burgess wrote Run Away for her brother and others who live with Duchenne muscular dystrophy to help raise awareness and funds to find a cure for this devastating disease. Burgess performed with Jack Wolf and the Broadview Heights High School marching band the Marching Bees in Ohio on October 10 and October 29. She also performed with the Big Red Marching Machine with the Illinois State University Band on November 8. I had the honor of singing "Run Away" with the amazing Illinois State University Band and the Broadview Heights high school Marching Bees to help raise funds and awareness for, said Burgess. Both groups didn't just play "Run Away" as just a song, they played it with heartfelt understanding of our situation. My heart was so touched and I am so thankful for the bands sharing their talent to help Duchenne families.

30 Clinical Stage Program Targeting Duchenne Thank you to Burgess, the Wolf family, the marching bands and all the fans in the stadium for supporting. News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

31 December 2014 Special Message from the Founder Featured in Local Media End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital BioMarin and Prosensa Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare- Disease Portfolio Debra Miller Debra Miller, Founder and CEO of, was featured in two local media outlets recently. Miller was highlighted as one of the top 10 people to watch in Newport Beach Magazine. Miller was also interviewed on the Friendship Show on KX93.5. Media Statement Regarding BioMarin and Prosensa Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD

32 Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into Clinical Stage Program Targeting News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

33 December 2014 Special Message from the Founder End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients Posture and Advanced Power Wheelchair Options for Those Living Duchenne Therapy Network and released their newest video blog that highlights posture and advanced power wheelchair options available for those living with Duchenne muscular dystrophy. Jennifer Wallace, founder of the Duchenne Therapy Network, and Kevin Phillips from Experea Healthcare discuss posture problems, benefits of custom seating and switch control options on power wheelchairs. Here are some highlights from the video. Common Posture Problems: Benefits of Custom Seating Switch Control Options Lateral curvature including scoliosis Lumbar lordosis arching the lower back Thoracic kyphosis slouching the upper back Good alignment Decrease skin breakdown Cool and dry interface Trunk stability for greater extremity use Power wheelchair mobility Seating functions Phone and table use Wireless computer mouse control Home environmental controls Please watch the video for more detailed information. Video blog 7 - Advanced Power Wheelchair Options- Duchen PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

34 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

35 December 2014 Special Message from the Founder End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare-Disease Portfolio Climb to, Fall 2014 Set your own personal climbing challenge big or small to help us conquer Duchenne. Do it yourself or put together a team and encourage your friends, family and colleagues to sponsor your climb. Click the blue "Create a Team" button above to start a team. Or to join a team already registered, click the "Join a Team" button above. Teams of people across the country will climb a mountain, hill or tall building to raise awareness and fund research to find a cure for Duchenne. This is the sixth annual Climb to. All funds will benefit. Pick Your Peak you can climb any mountain, hill or tall building. There are no geographic limitations. Anyone, regardless of athletic ability, is encouraged to participate individually or to form a team and secure personal sponsors/contributors for completing the climb. Choose a day that works best for you. Media Statement Regarding Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into

36 Clinical Stage Program Targeting Duchenne News Clay Matthews of the Green Bay Packers Featured in New PSA Napa in Newport Wine Auction Holiday Shopping at Amazon Smile Supports Recent Success Marching Bands Perform Run Away With Sarah Burgess Featured in Local Media Duchenne Therapy Network Posture and Advanced Power Wheelchair Options for Those Living Duchenne Upcoming Events Climb to, Fall 2014 Champions to, February 28, 2015, Newport Beach, Calif.

37 December 2014 Special Message from the Founder Champions to, Napa in Newport Wine Auction, February 28, 2015, Newport Beach, Calif. End of Year Giving to Sponsor Duplication for with Dr. Kevin Flanigan from Nationwide Children s Hospital BioMarin and Prosensa Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Products to Rare- Disease Portfolio Media Statement Regarding BioMarin and Prosensa Announcement Offers Continued Support to BioMarin for Duchenne Pfizer Initiates Phase 2 Study of PF in Reserve the date for the Champions to Napa in Newport Wine Auction on February 28, Once again this event will be held at the Balboa Bay Club in Newport Beach, Calif. For more information, click here. For sponsorship information, contact Drew Hoyer at or drew@cureduchenne.org. Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C110 in DMD Patients