Welcome & Global Genes Mission in Action

Transcription

1 Welcome & Global Genes Mission in Action Kym Kilbourne Vice President of Patient Advocacy, Global Genes Proudly supported by:

2 Global Genes Mission To Eliminate the Challenges of Rare Disease We develop resources and tools to help equip patient advocates to become successful ACTIVISTS for their disease We are working to build a global connected network a platform for collaboration and success We will fund Science and Technology innovations that will broadly impact patients within their lifetime

3 Advocacy & Education Global Genes Events and Programs

4 Foundation Alliance Launched in 2013 Growth A little more than 100 in 2013 Today more than 340 participating organizations Participation Advise on needs of the community, access Global Genes resources, greater connectivity with rare disease foundations and biotech companies Benefits Collaboration, networking and leadership opportunities, quarterly webinars, private FB page/members-only page, early registration access for the Summit, preferred room rates

5 RARE Educational Toolkits Online & print resources that help educate rare disease advocates about pressing topics critical to their journey. Collaboration Benefits Build understanding across stakeholders on key issues Topics Understanding Gene Therapy, Storytelling Through Social Media, How to Facilitate a Support Group, Strategies for Effective Fundraising

6 RARE Webinars RARE Webinars have been educating patient advocates, family members, and caregivers since January Collaboration Benefits Provide an opportunity for audience members to listen, collaborate and learn from from subject matter experts Topics Keys to Communicating About Rare Disease, Understanding Gene Therapy, Planning for Transition for an Adult Child with a Rare Disease, Grant Writing 101

7 David R. Cox Prize for RARE Compassion Overview Third year Collaboration with STAND Essay content for medical students Three Awards Announced at Summit Connect with MSAB Results in ,500 families requested matching 55 medical schools 65 rare diseases represented, 78 essays

8 We Are More incubated by GG Group of teens across rare and genetic disease space advocating to ensure teens living with chronic illness are viewed as more than their disease. Led by Shira Strongin Teen engagement in advocacy Social outreach Video storytelling Website

9 RARE Patient Impact Grant Program Funding nonprofits and support groups directly impacting rare disease patients 18 Grants awarded, range from $500 to $15,000 Sample Communities Awarded: CADISIL FA NGLY1 Sickle Cell 1p36 deletion PKU Funded by donations raised from Tribute to Champions and Denim Dash

10 RARE Patient Advocacy Summit 5th Annual RARE Patient Advocacy Summit September 22-23, 2016 Connect patient advocates Offers interactive discussions, insights and tools from leaders - advocacy organizations, government, industry, research & scientific Attending: 400+ attendees Up to 5,000 attending via Livestream

11 Tribute to Champions of Hope 5th Annual Tribute to Champions of Hope September 24, 2016 Unify the rare disease community and honor those individuals leading the fight against rare diseases & disorders. Attending 650 attendees Honored 15 individuals and organizations Including 200+ patient advocates Biotech & Pharmaceutical Executives, Physicians, Researchers, Healthcare Philanthropists, Celebrity Presenters, World Class Entertainment

12 Corporate Alliance Abeona Pharmaceuticals(*) Actelion Pharmaceuticals Aegerion Pharmaceuticals Alexion Pharmaceuticals Alnylam Pharmaceuticals Akcea Therapeutics Amicus Therapeutics Audentes Therapeutics Bayer Healthcare BIO Biogen Idec BioMarin Pharmaceutical Bluebird Bio Boehringer Ingelheim Caligor RX Cambridge BioMarketing Catalyst Pharma Cincinnati Children s Hospital Clementia Pharmaceuticals Connexion Healthcare Dicerna Discovery USA Dohmen Life Sciences Engage Health, Inc. Feinstein Kean Healthcare Genzyme Corporation GlaxoSmithKline Healthcare at Home Horizon Pharma Idis Limited Insmed Incorporated Intercept Pharma Invitae Isis Jazz Pharmaceuticals Janssen Lilly Lumos Pharma Marathon (*) Mallinckrodt (*) Medical Marketing Economics Novartis Pharmaceuticals Pfizer Inc. PTC Therapeutics(*) Quest Diagnostics(*) Raptor Pharmaceuticals Recombine Recordati Rare Diseases REGENXBIO(*) Retrophin Sarepta Therapuetics Shire Sigma-Tau Pharmaceuticals Siren Interactive SmithSolve Strongbridge Pharmaceuticals(*) Takeda Ultragenyx Vanda Pharmaceuticals, Inc. Vertex Pharmaceuticals, Inc. Vtesse(*) Walgreens

13 2016 Corporate Alliance Programs RARE University RAREMed Forums Annual Clinical Genetics Meeting (ACGM) March 9-11 in Tampa, FL Hosted first Patient Access Symposium Focused on barriers to accessing genetic testing

14 Contact the Global Genes Advocacy Team Kym Kilbourne Amanda Knitter Carrie Ostrea Ashley Yee Kym Amanda Carrie Ashley