NTSAD. all rare. all hopeful. all in common. all empowered. NTSADMission. Individually we are one drop. Together, we are an ocean.

Transcription

1 NTSAD Natioal Tay-Sachs & Allied Diseases Associatio Idividuy we are oe drop. Together, we are a ocea. Ryuosuke Satoro BOARD OF DIRECTORS SCIENTIFIC ADVISORY COMMITTEE Presidet Bria Maig Chairs Fraces Platt, PhD Jodi Hoffma, MD (Vice Chair) Vice Presidet Blyth Lord Treasurer Alex Wright Secretary Ruth Feldma Directors Merle Adelma Stewart Altma Risa Ase Jaye Gershkowitz Moica Gettlema Joh Gordo Scott Huger Meryl Kish Staci Kish Eda Kapla Amy Katz, Esq. Sophia Pesotchisky Kevi Romer Shari Ugerleider Mario Yaovsky Members Miriam Blitzer, PhD Robert Desick, PhD, MD Floria Eichler, MD Mark Haskis, VMD, MS, PhD Michael M. Kaback, MD, FACMG Edwi H. Kolody, MD Heather Lau, MD Paola Leoe, PhD Gustavo Maegawa, MD, PhD Marvi Natowicz, MD, PhD Swati Sathe, MD, MS Thomas N. Seyfried, PhD Barbara Shapiro, MD, PhD Eva Y. Syder, MD, PhD Cythia J. Tifft, MD, PhD, FACMG Steve U. Walkley, DVM, PhD Michael S. Watso, PhD, FACMG Past Presidets Shari Ugerleider Kevi Romer Thomas P. Lord Bradley L. Campbell Joh F. Crowley, JD Mark P. Madse Daiel N. Turer, Esq. Meredith R. Margolis, PhD Yvoe Sacks Daiel Greeberg, Esq. Sedra Schiffma Steve Laver, Esq.* Jaye Mackta Judith Saperstei Claire Kah Evely Sussma * deceased NTSADMissio NTSAD leads the fight to treat ad cure Tay-Sachs, Caava ad related geetic diseases, ad supports affected families ad idividuals i leadig fuller lives. rare. Natioal Tay-Sachs & Allied Diseases Associatio 2001 Beaco Street Suite 204 Bosto, Massachusetts (617) (800) 90-NTSAD hopeful. Executive Director Susa R. Kah Director of Developmet Joa Lawrece Director of Family Services Diaa Pagois Office Maager Igrid Miller Coferece Coordiator Becky Beso Developmet Associate Aliso Perkus CORPORATE ADVISORY COUNCIL Oved Amitay Ritu Baral Michael Gladstoe Kate Havilad Joa Keutzer, PhD Greg Licholai, MD i commo. empowered. Desig by Beth Bedavid-Val 2015 I ANNUAL REPORT

2 NTSAD Natioal Tay-Sachs & Allied Diseases Associatio Idividuy we are oe drop. Together, we are a ocea. Ryuosuke Satoro BOARD OF DIRECTORS SCIENTIFIC ADVISORY COMMITTEE Presidet Bria Maig Chairs Fraces Platt, PhD Jodi Hoffma, MD (Vice Chair) Vice Presidet Blyth Lord Treasurer Alex Wright Secretary Ruth Feldma Directors Merle Adelma Stewart Altma Risa Ase Jaye Gershkowitz Moica Gettlema Joh Gordo Scott Huger Meryl Kish Staci Kish Eda Kapla Amy Katz, Esq. Sophia Pesotchisky Kevi Romer Shari Ugerleider Mario Yaovsky Members Miriam Blitzer, PhD Robert Desick, PhD, MD Floria Eichler, MD Mark Haskis, VMD, MS, PhD Michael M. Kaback, MD, FACMG Edwi H. Kolody, MD Heather Lau, MD Paola Leoe, PhD Gustavo Maegawa, MD, PhD Marvi Natowicz, MD, PhD Swati Sathe, MD, MS Thomas N. Seyfried, PhD Barbara Shapiro, MD, PhD Eva Y. Syder, MD, PhD Cythia J. Tifft, MD, PhD, FACMG Steve U. Walkley, DVM, PhD Michael S. Watso, PhD, FACMG Past Presidets Shari Ugerleider Kevi Romer Thomas P. Lord Bradley L. Campbell Joh F. Crowley, JD Mark P. Madse Daiel N. Turer, Esq. Meredith R. Margolis, PhD Yvoe Sacks Daiel Greeberg, Esq. Sedra Schiffma Steve Laver, Esq.* Jaye Mackta Judith Saperstei Claire Kah Evely Sussma * deceased NTSADMissio NTSAD leads the fight to treat ad cure Tay-Sachs, Caava ad related geetic diseases, ad supports affected families ad idividuals i leadig fuller lives. rare. Natioal Tay-Sachs & Allied Diseases Associatio 2001 Beaco Street Suite 204 Bosto, Massachusetts (617) (800) 90-NTSAD hopeful. Executive Director Susa R. Kah Director of Developmet Joa Lawrece Director of Family Services Diaa Pagois Office Maager Igrid Miller Coferece Coordiator Becky Beso Developmet Associate Aliso Perkus CORPORATE ADVISORY COUNCIL Oved Amitay Ritu Baral Michael Gladstoe Kate Havilad Joa Keutzer, PhD Greg Licholai, MD i commo. empowered. Desig by Beth Bedavid-Val 2015 I ANNUAL REPORT

3 NTSADResearch NTSADFamilies NTSADVisio NTSADSupport NTSADAwareess 2015 NTSAD Sciece Symposium & Workshop for Professioals It is oly through NTSAD that we are able to fid To fud global cuttig-edge research, to provide families with compassioate care, ad to support ad collaborate effectively with the healthcare commuity to achieve our goals. It was a milestoe year for NTSAD because for the first time NTSAD reported reveues totalig over $1 millio i its 2015 fiscal year. It was possible thaks to the 2,735 doors who supported NTSAD ad who believe i the missio to care ad advocate for families, thoughtfully fud research i its stages, ad raise awareess about these rare geetic disease that affect too may. 21st Cetury Cures Defiig the Natural History of Caava Disease through Developmet of a Iteratioal Registry Pricipal Ivestigator: Heather Lau, MD, New York Uiversity Co-ivestigator: Paola Leoe, PhD, Rowa Uiversity Co-fuded with The Caava Foudatio Itravascular Gee Therapy for Felie GM2 Gagliosidosis Pricipal Ivestigator: Doug Marti, PhD Aubur Uiversity College of Veteriary Medicie Geeratio of a Kock-i Mutat HexB Mouse Model Pricipal Ivestigator: Eric Sjoberg, PhD OrPhi Therapeutics Pre-cliical Studies of AAVrh8-Hex Gee Delivery Pricipal Ivestigator: Miguel Sea-Esteves, PhD Uiversity of Massachusetts Medical Ceter family attedees thaks to a Helpig Had Grat award MICHAEL SUSSMAN, a logtime NTSAD supporter, received the NTSAD Above & Beyod award at the 2015 Aual Family Coferece for his geerous support of NTSAD. He cotiues to exted his parets legacy which started whe they, with other families, fouded NTSAD i the late 19s. Michael shared how proud his parets would be to see those i attedace there that day ad the work that they ad of NTSAD cotiues to do. Fifth Aual Day of Hope Ryig & Raisig Fuds for Research POWER OF ONE AWARD very September, the NTSAD E commuity ries behid Day of Hope as a way to be empowered ad raise fuds for research. I 2015, the Aual Day of Hope raised over $35,825 for research. Rare Sibligs Project This website project was ispired by a siblig ad brought to fruitio thaks to a Gezyme Patiet Advocacy (PAL) grat ad collaboratio betwee NTSAD ad Courageous Parets Network. Its purpose is to give sibligs a place to address the issues they face as rare sibligs ad share their stories with rare sibligs worldwide. 168, FAMILY EVENTS IN TOTAL UNAFFECTED % NTSAD Family Support FACEBOOK GROUP GERMANY KINGDOM MEXICO RAISED SINCE 2010 ISRAEL PHILIPPINES 439 MEMBERS HONDURAS BRAZIL $725,393 Ashley Watso held her aual Ride for Jase i memory of her so, Jase, raisig over $7,000 for Day of Hope i AFFECTED NTSAD ecourages meetig with a geetic couselor to uderstad your risks before gettig tested Fiscal Year udited Fiacial Statemets A $ ORALEA MARQUARDT, with her family, received the NTSAD Power of Oe award at NTSAD s 2015 Family Coferece for her cotributios to the developmet of a pediatric hospice i Florida, her commitmet to support other NTSAD families ad her work with her local hospice. Her so, William, who had GM-1, is her ispiratio ad guides everythig she has dcludig her pursuit of a Masters of Social Work degree. Carrier screeig remais our strogest weapo i the fight agaist diseases like Tay-Sachs, GM1, Sadhoff ad Caava. NTSAD recetly aouced its partership with JScree, Emory Uiversity, ad Cousyl, to fud ad coduct a research study that may provide essetial iformatio for the future trasitio of Tay-Sachs ezyme screeig to ewer geetic screeig testig methods. NTSAD together with the Mathew Forbes Romer Foudatio, Eva Lee Ugerleider Fud, New York Area Fud of NTSAD ad the Camero ad Hayde Lord Foudatio is fudig this importat study. of the geeral populatio is a carrier of the Tay-Sachs gee screeig 78FAMILIES Movig Forward to 1 i 190 Irish-Americas carrier This log weeked provides families with a carig eviromet that gives them the tools to feel empowered ad coected as they care or grieve for their loved oes. Over half the families who atted the coferece receive a Helpig bereaved families Had Grat award from a fud supported Late Oset adults plus by geerous doors, their exteded family foudatios ad caregivers, family, family fuds withi ewly parets or frieds ad diagosed spouses professioals NTSAD. Ashkeazi Jews, FrechCaadias, ad Cajus NEW ZEALAND NTSAD s 8th Aual Bosto beefit took place at the Royal Soesta i Cambridge, MA, o November 5th. A stellar silet auctio, ejoyable food, ad a woderful mix of frieds, parters ad families made for a successful eveig raisig over $80,000 for research ad support for NTSAD. Guests heard from a local NTSAD mom, Kate Hubbard who shared her story about carig for daughter Brook ad the rest of her family durig Brook s too short life livig with Tay-Sachs. It was also a hoor to recogize the work of Bob Coughli, Presidet ad CEO of Massachusetts Biotechology Coucil ad to liste to his story about his persoal coectio with rare diseases ad his cotributio to the rare disease commuity. The 21st Cetury Cures Act made it through the House i 2015 ad ow waits its tur o the Seate floor. This bill could accelerate the pace of discovery, developmet, ad delivery of rare disease treatmets ad cures, as well as provide hope for patiets ad their families. Through parterig with the Natioal Orgaizatio for Rare Disorders, EveryLife Foudatio ad Global Gees, NTSAD brigs ews of the latest rare disease legislatio to its families so they ca coect with their local represetatives to put a face o rare diseases. FRANCE Registry ad repository for Late Oset GM2 gagliosidoses Pricipal Ivestigator: Floria Eichler, MD Massachusetts Geeral Hospital Imagie & Believe Aual Family Coferece April 2015, Resto, Virgiia 2015 Research Iitiative Grats Awarded Developmet ad validatio of a rapid, MS/MS-based method to detect Hexosamiodase deficiecy i Tay-Sachs disease Pricipal Ivestigator: Deis Lehotay, PhD Quees Uiversity, Otario ABOVE AND BEYOND AWARD % Research 32% Family Services 12% Fudraisig 10% Educatio 6% Admiistratio 3% Advocacy STATES CANADA AUSTRALIA $1,106,524 SUPPORT & REVENUE Team NTSAD rode to raise $20,000 o May 8 i Philadelphia which was matched by UPe s Orpha Disease Ceter to support a grat award made to Marlee Jacobso, PhD, of Temple Uiversity. Her project, Patiet-Derived Pheotypic Assay to Discover Treatmets for Tay-Sachs disease, will eable exploratio of sm molecules icludig kow FDAapproved drugs that, if prove to be effective, could have the potetial for rapid progressio ito cliical trials. The heart ad soul of NTSAD is our family services program. We advocate for childre affected by Tay-Sachs, GM1, Sadhoff ad Caava diseases, coect parets copig with the heartache ad support the adults strugglig with the degeerative effects of the Late Oset forms. Each ad every perso coectig with NTSAD will fid solace ad ivaluable support withi this sm but mighty commuity. KNOW THE FACTS: ARGENTINA Secod Aual Millio Dollar Bike Ride other families to coect with ad help us o our jourey. I caot fathom doig this aloe. EXPENSES NTSAD brought healthcare professioals ad researchers together to discuss topics from basic sciece to cliical trial readiess for a symposium precedig the 2015 NTSAD Aual Family Coferece. They gathered together to develop ways i which we ca parter to make progress. Topics explored raged from lookig at the commo elemets amog these eurodegeerative diseases to cliical trial desig to specific issues relatig to the gagliosidoses ad leukodystrophies. 2 36% Research 17% Restricted Gifts 15% Gifts 11% Coferece 10% Special Evets 8% Grats 3% Quality Cotrol

4 NTSADResearch NTSADFamilies NTSADVisio NTSADSupport NTSADAwareess 2015 NTSAD Sciece Symposium & Workshop for Professioals It is oly through NTSAD that we are able to fid To fud global cuttig-edge research, to provide families with compassioate care, ad to support ad collaborate effectively with the healthcare commuity to achieve our goals. It was a milestoe year for NTSAD because for the first time NTSAD reported reveues totalig over $1 millio i its 2015 fiscal year. It was possible thaks to the 2,735 doors who supported NTSAD ad who believe i the missio to care ad advocate for families, thoughtfully fud research i its stages, ad raise awareess about these rare geetic disease that affect too may. 21st Cetury Cures Defiig the Natural History of Caava Disease through Developmet of a Iteratioal Registry Pricipal Ivestigator: Heather Lau, MD, New York Uiversity Co-ivestigator: Paola Leoe, PhD, Rowa Uiversity Co-fuded with The Caava Foudatio Itravascular Gee Therapy for Felie GM2 Gagliosidosis Pricipal Ivestigator: Doug Marti, PhD Aubur Uiversity College of Veteriary Medicie Geeratio of a Kock-i Mutat HexB Mouse Model Pricipal Ivestigator: Eric Sjoberg, PhD OrPhi Therapeutics Pre-cliical Studies of AAVrh8-Hex Gee Delivery Pricipal Ivestigator: Miguel Sea-Esteves, PhD Uiversity of Massachusetts Medical Ceter family attedees thaks to a Helpig Had Grat award MICHAEL SUSSMAN, a logtime NTSAD supporter, received the NTSAD Above & Beyod award at the 2015 Aual Family Coferece for his geerous support of NTSAD. He cotiues to exted his parets legacy which started whe they, with other families, fouded NTSAD i the late 19s. Michael shared how proud his parets would be to see those i attedace there that day ad the work that they ad of NTSAD cotiues to do. Fifth Aual Day of Hope Ryig & Raisig Fuds for Research POWER OF ONE AWARD very September, the NTSAD E commuity ries behid Day of Hope as a way to be empowered ad raise fuds for research. I 2015, the Aual Day of Hope raised over $35,825 for research. Rare Sibligs Project This website project was ispired by a siblig ad brought to fruitio thaks to a Gezyme Patiet Advocacy (PAL) grat ad collaboratio betwee NTSAD ad Courageous Parets Network. Its purpose is to give sibligs a place to address the issues they face as rare sibligs ad share their stories with rare sibligs worldwide. 168, FAMILY EVENTS IN TOTAL UNAFFECTED % NTSAD Family Support FACEBOOK GROUP GERMANY KINGDOM MEXICO RAISED SINCE 2010 ISRAEL PHILIPPINES 439 MEMBERS HONDURAS BRAZIL $725,393 Ashley Watso held her aual Ride for Jase i memory of her so, Jase, raisig over $7,000 for Day of Hope i AFFECTED NTSAD ecourages meetig with a geetic couselor to uderstad your risks before gettig tested Fiscal Year udited Fiacial Statemets A $ ORALEA MARQUARDT, with her family, received the NTSAD Power of Oe award at NTSAD s 2015 Family Coferece for her cotributios to the developmet of a pediatric hospice i Florida, her commitmet to support other NTSAD families ad her work with her local hospice. Her so, William, who had GM-1, is her ispiratio ad guides everythig she has dcludig her pursuit of a Masters of Social Work degree. Carrier screeig remais our strogest weapo i the fight agaist diseases like Tay-Sachs, GM1, Sadhoff ad Caava. NTSAD recetly aouced its partership with JScree, Emory Uiversity, ad Cousyl, to fud ad coduct a research study that may provide essetial iformatio for the future trasitio of Tay-Sachs ezyme screeig to ewer geetic screeig testig methods. NTSAD together with the Mathew Forbes Romer Foudatio, Eva Lee Ugerleider Fud, New York Area Fud of NTSAD ad the Camero ad Hayde Lord Foudatio is fudig this importat study. of the geeral populatio is a carrier of the Tay-Sachs gee screeig 78FAMILIES Movig Forward to 1 i 190 Irish-Americas carrier This log weeked provides families with a carig eviromet that gives them the tools to feel empowered ad coected as they care or grieve for their loved oes. Over half the families who atted the coferece receive a Helpig bereaved families Had Grat award from a fud supported Late Oset adults plus by geerous doors, their exteded family foudatios ad caregivers, family, family fuds withi ewly parets or frieds ad diagosed spouses professioals NTSAD. Ashkeazi Jews, FrechCaadias, ad Cajus NEW ZEALAND NTSAD s 8th Aual Bosto beefit took place at the Royal Soesta i Cambridge, MA, o November 5th. A stellar silet auctio, ejoyable food, ad a woderful mix of frieds, parters ad families made for a successful eveig raisig over $80,000 for research ad support for NTSAD. Guests heard from a local NTSAD mom, Kate Hubbard who shared her story about carig for daughter Brook ad the rest of her family durig Brook s too short life livig with Tay-Sachs. It was also a hoor to recogize the work of Bob Coughli, Presidet ad CEO of Massachusetts Biotechology Coucil ad to liste to his story about his persoal coectio with rare diseases ad his cotributio to the rare disease commuity. The 21st Cetury Cures Act made it through the House i 2015 ad ow waits its tur o the Seate floor. This bill could accelerate the pace of discovery, developmet, ad delivery of rare disease treatmets ad cures, as well as provide hope for patiets ad their families. Through parterig with the Natioal Orgaizatio for Rare Disorders, EveryLife Foudatio ad Global Gees, NTSAD brigs ews of the latest rare disease legislatio to its families so they ca coect with their local represetatives to put a face o rare diseases. FRANCE Registry ad repository for Late Oset GM2 gagliosidoses Pricipal Ivestigator: Floria Eichler, MD Massachusetts Geeral Hospital Imagie & Believe Aual Family Coferece April 2015, Resto, Virgiia 2015 Research Iitiative Grats Awarded Developmet ad validatio of a rapid, MS/MS-based method to detect Hexosamiodase deficiecy i Tay-Sachs disease Pricipal Ivestigator: Deis Lehotay, PhD Quees Uiversity, Otario ABOVE AND BEYOND AWARD % Research 32% Family Services 12% Fudraisig 10% Educatio 6% Admiistratio 3% Advocacy STATES CANADA AUSTRALIA $1,106,524 SUPPORT & REVENUE Team NTSAD rode to raise $20,000 o May 8 i Philadelphia which was matched by UPe s Orpha Disease Ceter to support a grat award made to Marlee Jacobso, PhD, of Temple Uiversity. Her project, Patiet-Derived Pheotypic Assay to Discover Treatmets for Tay-Sachs disease, will eable exploratio of sm molecules icludig kow FDAapproved drugs that, if prove to be effective, could have the potetial for rapid progressio ito cliical trials. The heart ad soul of NTSAD is our family services program. We advocate for childre affected by Tay-Sachs, GM1, Sadhoff ad Caava diseases, coect parets copig with the heartache ad support the adults strugglig with the degeerative effects of the Late Oset forms. Each ad every perso coectig with NTSAD will fid solace ad ivaluable support withi this sm but mighty commuity. KNOW THE FACTS: ARGENTINA Secod Aual Millio Dollar Bike Ride other families to coect with ad help us o our jourey. I caot fathom doig this aloe. EXPENSES NTSAD brought healthcare professioals ad researchers together to discuss topics from basic sciece to cliical trial readiess for a symposium precedig the 2015 NTSAD Aual Family Coferece. They gathered together to develop ways i which we ca parter to make progress. Topics explored raged from lookig at the commo elemets amog these eurodegeerative diseases to cliical trial desig to specific issues relatig to the gagliosidoses ad leukodystrophies. 2 36% Research 17% Restricted Gifts 15% Gifts 11% Coferece 10% Special Evets 8% Grats 3% Quality Cotrol

5 NTSADResearch NTSADFamilies NTSADVisio NTSADSupport NTSADAwareess 2015 NTSAD Sciece Symposium & Workshop for Professioals It is oly through NTSAD that we are able to fid To fud global cuttig-edge research, to provide families with compassioate care, ad to support ad collaborate effectively with the healthcare commuity to achieve our goals. It was a milestoe year for NTSAD because for the first time NTSAD reported reveues totalig over $1 millio i its 2015 fiscal year. It was possible thaks to the 2,735 doors who supported NTSAD ad who believe i the missio to care ad advocate for families, thoughtfully fud research i its stages, ad raise awareess about these rare geetic disease that affect too may. 21st Cetury Cures Defiig the Natural History of Caava Disease through Developmet of a Iteratioal Registry Pricipal Ivestigator: Heather Lau, MD, New York Uiversity Co-ivestigator: Paola Leoe, PhD, Rowa Uiversity Co-fuded with The Caava Foudatio Itravascular Gee Therapy for Felie GM2 Gagliosidosis Pricipal Ivestigator: Doug Marti, PhD Aubur Uiversity College of Veteriary Medicie Geeratio of a Kock-i Mutat HexB Mouse Model Pricipal Ivestigator: Eric Sjoberg, PhD OrPhi Therapeutics Pre-cliical Studies of AAVrh8-Hex Gee Delivery Pricipal Ivestigator: Miguel Sea-Esteves, PhD Uiversity of Massachusetts Medical Ceter family attedees thaks to a Helpig Had Grat award MICHAEL SUSSMAN, a logtime NTSAD supporter, received the NTSAD Above & Beyod award at the 2015 Aual Family Coferece for his geerous support of NTSAD. He cotiues to exted his parets legacy which started whe they, with other families, fouded NTSAD i the late 19s. Michael shared how proud his parets would be to see those i attedace there that day ad the work that they ad of NTSAD cotiues to do. Fifth Aual Day of Hope Ryig & Raisig Fuds for Research POWER OF ONE AWARD very September, the NTSAD E commuity ries behid Day of Hope as a way to be empowered ad raise fuds for research. I 2015, the Aual Day of Hope raised over $35,825 for research. Rare Sibligs Project This website project was ispired by a siblig ad brought to fruitio thaks to a Gezyme Patiet Advocacy (PAL) grat ad collaboratio betwee NTSAD ad Courageous Parets Network. Its purpose is to give sibligs a place to address the issues they face as rare sibligs ad share their stories with rare sibligs worldwide. 168, FAMILY EVENTS IN TOTAL UNAFFECTED % NTSAD Family Support FACEBOOK GROUP GERMANY KINGDOM MEXICO RAISED SINCE 2010 ISRAEL PHILIPPINES 439 MEMBERS HONDURAS BRAZIL $725,393 Ashley Watso held her aual Ride for Jase i memory of her so, Jase, raisig over $7,000 for Day of Hope i AFFECTED NTSAD ecourages meetig with a geetic couselor to uderstad your risks before gettig tested Fiscal Year udited Fiacial Statemets A $ ORALEA MARQUARDT, with her family, received the NTSAD Power of Oe award at NTSAD s 2015 Family Coferece for her cotributios to the developmet of a pediatric hospice i Florida, her commitmet to support other NTSAD families ad her work with her local hospice. Her so, William, who had GM-1, is her ispiratio ad guides everythig she has dcludig her pursuit of a Masters of Social Work degree. Carrier screeig remais our strogest weapo i the fight agaist diseases like Tay-Sachs, GM1, Sadhoff ad Caava. NTSAD recetly aouced its partership with JScree, Emory Uiversity, ad Cousyl, to fud ad coduct a research study that may provide essetial iformatio for the future trasitio of Tay-Sachs ezyme screeig to ewer geetic screeig testig methods. NTSAD together with the Mathew Forbes Romer Foudatio, Eva Lee Ugerleider Fud, New York Area Fud of NTSAD ad the Camero ad Hayde Lord Foudatio is fudig this importat study. of the geeral populatio is a carrier of the Tay-Sachs gee screeig 78FAMILIES Movig Forward to 1 i 190 Irish-Americas carrier This log weeked provides families with a carig eviromet that gives them the tools to feel empowered ad coected as they care or grieve for their loved oes. Over half the families who atted the coferece receive a Helpig bereaved families Had Grat award from a fud supported Late Oset adults plus by geerous doors, their exteded family foudatios ad caregivers, family, family fuds withi ewly parets or frieds ad diagosed spouses professioals NTSAD. Ashkeazi Jews, FrechCaadias, ad Cajus NEW ZEALAND NTSAD s 8th Aual Bosto beefit took place at the Royal Soesta i Cambridge, MA, o November 5th. A stellar silet auctio, ejoyable food, ad a woderful mix of frieds, parters ad families made for a successful eveig raisig over $80,000 for research ad support for NTSAD. Guests heard from a local NTSAD mom, Kate Hubbard who shared her story about carig for daughter Brook ad the rest of her family durig Brook s too short life livig with Tay-Sachs. It was also a hoor to recogize the work of Bob Coughli, Presidet ad CEO of Massachusetts Biotechology Coucil ad to liste to his story about his persoal coectio with rare diseases ad his cotributio to the rare disease commuity. The 21st Cetury Cures Act made it through the House i 2015 ad ow waits its tur o the Seate floor. This bill could accelerate the pace of discovery, developmet, ad delivery of rare disease treatmets ad cures, as well as provide hope for patiets ad their families. Through parterig with the Natioal Orgaizatio for Rare Disorders, EveryLife Foudatio ad Global Gees, NTSAD brigs ews of the latest rare disease legislatio to its families so they ca coect with their local represetatives to put a face o rare diseases. FRANCE Registry ad repository for Late Oset GM2 gagliosidoses Pricipal Ivestigator: Floria Eichler, MD Massachusetts Geeral Hospital Imagie & Believe Aual Family Coferece April 2015, Resto, Virgiia 2015 Research Iitiative Grats Awarded Developmet ad validatio of a rapid, MS/MS-based method to detect Hexosamiodase deficiecy i Tay-Sachs disease Pricipal Ivestigator: Deis Lehotay, PhD Quees Uiversity, Otario ABOVE AND BEYOND AWARD % Research 32% Family Services 12% Fudraisig 10% Educatio 6% Admiistratio 3% Advocacy STATES CANADA AUSTRALIA $1,106,524 SUPPORT & REVENUE Team NTSAD rode to raise $20,000 o May 8 i Philadelphia which was matched by UPe s Orpha Disease Ceter to support a grat award made to Marlee Jacobso, PhD, of Temple Uiversity. Her project, Patiet-Derived Pheotypic Assay to Discover Treatmets for Tay-Sachs disease, will eable exploratio of sm molecules icludig kow FDAapproved drugs that, if prove to be effective, could have the potetial for rapid progressio ito cliical trials. The heart ad soul of NTSAD is our family services program. We advocate for childre affected by Tay-Sachs, GM1, Sadhoff ad Caava diseases, coect parets copig with the heartache ad support the adults strugglig with the degeerative effects of the Late Oset forms. Each ad every perso coectig with NTSAD will fid solace ad ivaluable support withi this sm but mighty commuity. KNOW THE FACTS: ARGENTINA Secod Aual Millio Dollar Bike Ride other families to coect with ad help us o our jourey. I caot fathom doig this aloe. EXPENSES NTSAD brought healthcare professioals ad researchers together to discuss topics from basic sciece to cliical trial readiess for a symposium precedig the 2015 NTSAD Aual Family Coferece. They gathered together to develop ways i which we ca parter to make progress. Topics explored raged from lookig at the commo elemets amog these eurodegeerative diseases to cliical trial desig to specific issues relatig to the gagliosidoses ad leukodystrophies. 2 36% Research 17% Restricted Gifts 15% Gifts 11% Coferece 10% Special Evets 8% Grats 3% Quality Cotrol

6 NTSAD Natioal Tay-Sachs & Allied Diseases Associatio Idividuy we are oe drop. Together, we are a ocea. Ryuosuke Satoro BOARD OF DIRECTORS SCIENTIFIC ADVISORY COMMITTEE Presidet Bria Maig Chairs Fraces Platt, PhD Jodi Hoffma, MD (Vice Chair) Vice Presidet Blyth Lord Treasurer Alex Wright Secretary Ruth Feldma Directors Merle Adelma Stewart Altma Risa Ase Jaye Gershkowitz Moica Gettlema Joh Gordo Scott Huger Meryl Kish Staci Kish Eda Kapla Amy Katz, Esq. Sophia Pesotchisky Kevi Romer Shari Ugerleider Mario Yaovsky Members Miriam Blitzer, PhD Robert Desick, PhD, MD Floria Eichler, MD Mark Haskis, VMD, MS, PhD Michael M. Kaback, MD, FACMG Edwi H. Kolody, MD Heather Lau, MD Paola Leoe, PhD Gustavo Maegawa, MD, PhD Marvi Natowicz, MD, PhD Swati Sathe, MD, MS Thomas N. Seyfried, PhD Barbara Shapiro, MD, PhD Eva Y. Syder, MD, PhD Cythia J. Tifft, MD, PhD, FACMG Steve U. Walkley, DVM, PhD Michael S. Watso, PhD, FACMG Past Presidets Shari Ugerleider Kevi Romer Thomas P. Lord Bradley L. Campbell Joh F. Crowley, JD Mark P. Madse Daiel N. Turer, Esq. Meredith R. Margolis, PhD Yvoe Sacks Daiel Greeberg, Esq. Sedra Schiffma Steve Laver, Esq.* Jaye Mackta Judith Saperstei Claire Kah Evely Sussma * deceased NTSADMissio NTSAD leads the fight to treat ad cure Tay-Sachs, Caava ad related geetic diseases, ad supports affected families ad idividuals i leadig fuller lives. rare. Natioal Tay-Sachs & Allied Diseases Associatio 2001 Beaco Street Suite 204 Bosto, Massachusetts (617) (800) 90-NTSAD hopeful. Executive Director Susa R. Kah Director of Developmet Joa Lawrece Director of Family Services Diaa Pagois Office Maager Igrid Miller Coferece Coordiator Becky Beso Developmet Associate Aliso Perkus CORPORATE ADVISORY COUNCIL Oved Amitay Ritu Baral Michael Gladstoe Kate Havilad Joa Keutzer, PhD Greg Licholai, MD i commo. empowered. Desig by Beth Bedavid-Val 2015 I ANNUAL REPORT