NTSAD. Spotlight on Imagine & Believe. NTSADMission

Transcription

1 NTSAD Spotlight o Imagie & Believe NTSAD recogized its 60th aiversary throughout 2017 by hoorig those families ad idividuals who established ad expaded the NTSAD commuity, by recogizig our preset achievemets, ad by embracig goals for the future i the areas of family support, research advaces, NTSAD celebrated its 60th Aiversary Imagie & Believe evet at the Royal Soesta i Cambridge o Thursday, November 9th. Highlights of the ight icluded a thoughtful discussio with hoorees Dr. David Meeker ad Professor Timothy Cox. Moderated by NTSAD s former Executive Director, Jaye Gershkowitz, topics icluded what motivates their work i the rare disease commuity, scietific progressios, ad the value that a orgaizatio like NTSAD ca have o both families ad supporters. This discussio was followed by heartfelt remarks by Talia s mother ad NTSAD paret, Carla Steckma. Joiig Carla ad hoorees, David ad Tim, were 190 guests who raised over $120,000 the evet s largest total sice its iceptio! BOARD OF DIRECTORS SCIENTIFIC ADVISORY COMMITTEE Presidet Blyth Lord Chairs Fraces Platt, PhD Jodi Hoffma, MD (Vice Chair) Vice Presidet Staci Kallish, DO Treasurer Alex Wright Secretary Ruth Feldma Directors Merle Adelma Stewart Altma Risa Ase Moica Gettlema Kare Grizaid William Johso, PhD Meryl Kallish Staci Kallish Eda Kapla Amy Katz, Esq. Sophia Pesotchisky Alec Rubestei Amy Speak Mario Yaovsky Past Presidets Bria Maig 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* Ruth Dukell* 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 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 Corporate Advisory Coucil Oved Amitay Ritu Baral Laura Cohe Joh Gordo Michael Gladstoe Kate Havilad Walt Kowtoiuk, PhD Greg Licholai, MD Joa Keutzer, PhD NTSADMissio Natioal Tay-Sachs & Allied Diseases Associatio NTSAD leads the fight to treat ad cure Tay-Sachs, Sadhoff, GM1, Caava ad related geetic diseases, ad supports affected families ad idividuals i leadig fuller lives. Natioal Tay-Sachs & Allied Diseases Associatio 2001 Beaco Street Suite 204 Bosto, Massachusetts (617) (800) 90-NTSAD Executive Director Susa R. Kah Director of Developmet Patrick Woods Director of Family Services Diaa Pagois Developmet & Admiistrative Associate Tsitsi Moyo Coferece Coordiator Becky Beso * deceased ad prevetative outreach ad educatio. Desig by Beth Bedavid-Val 2017 I ANNUAL REPORT

2 NTSAD Spotlight o Imagie & Believe NTSAD recogized its 60th aiversary throughout 2017 by hoorig those families ad idividuals who established ad expaded the NTSAD commuity, by recogizig our preset achievemets, ad by embracig goals for the future i the areas of family support, research advaces, NTSAD celebrated its 60th Aiversary Imagie & Believe evet at the Royal Soesta i Cambridge o Thursday, November 9th. Highlights of the ight icluded a thoughtful discussio with hoorees Dr. David Meeker ad Professor Timothy Cox. Moderated by NTSAD s former Executive Director, Jaye Gershkowitz, topics icluded what motivates their work i the rare disease commuity, scietific progressios, ad the value that a orgaizatio like NTSAD ca have o both families ad supporters. This discussio was followed by heartfelt remarks by Talia s mother ad NTSAD paret, Carla Steckma. Joiig Carla ad hoorees, David ad Tim, were 190 guests who raised over $120,000 the evet s largest total sice its iceptio! BOARD OF DIRECTORS SCIENTIFIC ADVISORY COMMITTEE Presidet Blyth Lord Chairs Fraces Platt, PhD Jodi Hoffma, MD (Vice Chair) Vice Presidet Staci Kallish, DO Treasurer Alex Wright Secretary Ruth Feldma Directors Merle Adelma Stewart Altma Risa Ase Moica Gettlema Kare Grizaid William Johso, PhD Meryl Kallish Staci Kallish Eda Kapla Amy Katz, Esq. Sophia Pesotchisky Alec Rubestei Amy Speak Mario Yaovsky Past Presidets Bria Maig 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* Ruth Dukell* 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 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 Corporate Advisory Coucil Oved Amitay Ritu Baral Laura Cohe Joh Gordo Michael Gladstoe Kate Havilad Walt Kowtoiuk, PhD Greg Licholai, MD Joa Keutzer, PhD NTSADMissio Natioal Tay-Sachs & Allied Diseases Associatio NTSAD leads the fight to treat ad cure Tay-Sachs, Sadhoff, GM1, Caava ad related geetic diseases, ad supports affected families ad idividuals i leadig fuller lives. Natioal Tay-Sachs & Allied Diseases Associatio 2001 Beaco Street Suite 204 Bosto, Massachusetts (617) (800) 90-NTSAD Executive Director Susa R. Kah Director of Developmet Patrick Woods Director of Family Services Diaa Pagois Developmet & Admiistrative Associate Tsitsi Moyo Coferece Coordiator Becky Beso * deceased ad prevetative outreach ad educatio. Desig by Beth Bedavid-Val 2017 I ANNUAL REPORT

3 2017 Research Iitiative Grats Awarded NTSAD partered with Caava Research Illiois ad Cure GM1 Foudatio o Patiet Isights Networks (PIN) for Caava ad GM1 diseases. The PIN s purpose is to allow families to eter iformatio about their disease experiece that is traslated ito deidetified data to pait a picture of the disease experiece across a global commuity. The data will ultimately help promisig research advace to cliical trials by bolsterig the hard data of the research. Proof of cocept study of HSC gee therapy for Tay-Sachs disease NTSAD ad Cure Tay-Sachs Foudatio (CTSF) have partered to lauch a GM2 Tay-Sachs ad Sadhoff Disease Patiet Isights Network (PIN) this sprig. Millio Dollar Bike Rides Raise $40,000 for NTSAD Grat Team NTSAD raised $40,000 from the Fourth Aual Millio Dollar Bike Ride held i Philadelphia, ad the amout was matched by UPe s Orpha Disease Ceter! The 2017 grat wet to Igor Nestrasil, MD, PhD, at the Uiversity of Miesota for his project Brai MRI sigatures i ifats with ifatile forms of GM-1 ad GM-2. Pricipal Ivestigator: Alessadra Biffi, MD Daa-Farber/Bosto Childre s Cacer ad Blood Disorders Ceter Miimally ivasive delivery of AAV gee therapy i the Tay-Sachs Sheep Pricipal Ivestigator: Heather Gray-Edwards, PhD Scott-Ritchey Research Ceter, Aubur Uiversity, Aubur, AL Developmet of a quatitative method for the determiatio of a petasaccharide i GM1-gagliosidosis patiet cells to assess the potetial therapeutic efficacy of a beta-galactosidase pharmacological chaperoe drug cadidate. Pricipal Ivestigators: Tim Wood, PhD, Greewood Geetic Ceter, Greewood, South Carolia ad Stephae Demotz, PhD, Dorpha, Lausae, Switzerlad Accelerated program for CSF delivery of AAV gee therapy for Tay-Sachs ad Sadhoff patiets Pricipal Ivestigator: Miguel Sea-Esteves, PhD Uiversity of Massachusetts Medical School Progress i Gee Therapy Research The Tay-Sachs Gee Therapy Cosortium completed aimal studies ad the follow-up pathology studies. The results met the criteria to advace to the ext stage. They will ext maufacture cliical grade vectors with a goal i 2018 to submit a Ivestigatioal New Drug (IND) filig to start cliical trials. NTSADAwareess NTSAD s ANNUAL FAMILY CONFERENCE: A Commuity of Support Expaded Carrier Screeig Progress was made i carrier screeig through the Cousyl JScree research project, The Hexosamiidase A Variats of Ukow Sigificace (HAVUS). The goal of this project was to reclassify commo HEXA VUS to likely beig/beig, thereby coferrig a higher carrier detectio rate for sequecig. This research will be a meaigful milestoe i the evolutio for Tay-Sachs carrier screeig from ezyme aalysis to sequecig. The Aual Family Coferece is the corerstoe of NTSAD s Family Services program. Families impacted by all forms of Tay-Sachs, Sadhoff, GM1 ad Caava diseases come together to support ad empower each other through similar ad shared experieces. The program ecompasses all facets of livig with these diseases from peer support, research updates, care maagemet ad choices, to a focus o relatioships, siblig support ad how to be a advocate. 3 FULL DAYS OVER 50,000 $ IN HELPING HANDS GRANTS give to help families atted the 2017 Aual Family Coferece This project was fuded by NTSAD ad affiliate family foudatios. KNOW THE FACTS: 40+ Ashkeazi Jews, Frech-Caadias, ad Cajus... FAMILIES HELPED New Support Resources to 1 i 190 Irish-Americas... Care Tip Series for Families The first two videos of the series lauched i the summer of 2017 to educate families o issues relatig to carig for a child. The topics covered G-Tubes A to Z ad Motio & Massage. The series will expad i the summer of 2018 to iclude two more topics relatig to care. More tha 20 evets ad over a doze custom t-shirt campaigs Families rallied ad raised fuds for research from coast to coast icludig Caada 50% AFFECTED 2017 Fiscal Year udited Fiacial Statemets A Emma s Fud for Families This ew fud, started by the Artiia family i memory of their daughter, Emma, was established to allow affected families to apply for grats to use towards erichmet activities. It ca be used ay way a family feels would be beeficial for creatig their memories, such as trips to the zoo, photo shoots, tickets to a evet, a overight i a hotel with a pool, etc. Commuity Rallies aroud Family Over $101,000 raised, brigig the total to over $320,000 sice 2011 UNAFFECTED NTSAD ecourages meetig with a geetic couselor to uderstad your risks before gettig tested. of the geeral populatio... NTSADEvets 2017 Day of Hope: Families Drive Research... is a carrier of the Tay-Sachs gee. screeig Traslatig Patiet Experiece to Help Research NTSADFamily carrier NTSADResearch We would like to give a special thaks to the Roaldso family ad frieds for raisig over $50,000 for research with their Day of Hope evet. Mollie ad Madely, who have Juveile Sadhoff, are a extraordiary ispiratio to ot oly their fried ad frieds, but to families worldwide. SUPPORT & REVENUE EXPENSES $809,635 $711,679 31% Research NTSADAdvocacy NTSAD, with ispiratio ad guidace from the EveryLife Foudatio ad the Natioal Orgaizatio for Rare Disorders, cotiued to ecourage families to reach out to their represetatives throughout the year. Sharig their stories ca stress the importace of votig for legislatio that ca eable families to be whole ad healthy. IRemembrace Jaso Arbetter Seaby Clark Jeter DeJog Ava Demeshko Cristia Feradez Avi Forrester Cash Gray Kylie Hatchel Amelie Lewi Levi Lucero, Jr. Cora McDoald Jea McKizie Alyssa Mushi Decla O Coell Ia Ortega Rio Page Mackezie Plick Barro Posey Cooper Richards Josiah Scaparotti Talia Steckma Nikko Zollar Jauary 1, 2017 December 31, % Gifts 18% Coferece 13% Special Evets 9% Grats 8% Restricted Gifts 1% Other 37% Research 34% Family Services 12% Fudraisig 7% Educatio 7% Admiistratio 3% Advocacy

4 2017 Research Iitiative Grats Awarded NTSAD partered with Caava Research Illiois ad Cure GM1 Foudatio o Patiet Isights Networks (PIN) for Caava ad GM1 diseases. The PIN s purpose is to allow families to eter iformatio about their disease experiece that is traslated ito deidetified data to pait a picture of the disease experiece across a global commuity. The data will ultimately help promisig research advace to cliical trials by bolsterig the hard data of the research. Proof of cocept study of HSC gee therapy for Tay-Sachs disease NTSAD ad Cure Tay-Sachs Foudatio (CTSF) have partered to lauch a GM2 Tay-Sachs ad Sadhoff Disease Patiet Isights Network (PIN) this sprig. Millio Dollar Bike Rides Raise $40,000 for NTSAD Grat Team NTSAD raised $40,000 from the Fourth Aual Millio Dollar Bike Ride held i Philadelphia, ad the amout was matched by UPe s Orpha Disease Ceter! The 2017 grat wet to Igor Nestrasil, MD, PhD, at the Uiversity of Miesota for his project Brai MRI sigatures i ifats with ifatile forms of GM-1 ad GM-2. Pricipal Ivestigator: Alessadra Biffi, MD Daa-Farber/Bosto Childre s Cacer ad Blood Disorders Ceter Miimally ivasive delivery of AAV gee therapy i the Tay-Sachs Sheep Pricipal Ivestigator: Heather Gray-Edwards, PhD Scott-Ritchey Research Ceter, Aubur Uiversity, Aubur, AL Developmet of a quatitative method for the determiatio of a petasaccharide i GM1-gagliosidosis patiet cells to assess the potetial therapeutic efficacy of a beta-galactosidase pharmacological chaperoe drug cadidate. Pricipal Ivestigators: Tim Wood, PhD, Greewood Geetic Ceter, Greewood, South Carolia ad Stephae Demotz, PhD, Dorpha, Lausae, Switzerlad Accelerated program for CSF delivery of AAV gee therapy for Tay-Sachs ad Sadhoff patiets Pricipal Ivestigator: Miguel Sea-Esteves, PhD Uiversity of Massachusetts Medical School Progress i Gee Therapy Research The Tay-Sachs Gee Therapy Cosortium completed aimal studies ad the follow-up pathology studies. The results met the criteria to advace to the ext stage. They will ext maufacture cliical grade vectors with a goal i 2018 to submit a Ivestigatioal New Drug (IND) filig to start cliical trials. NTSADAwareess NTSAD s ANNUAL FAMILY CONFERENCE: A Commuity of Support Expaded Carrier Screeig Progress was made i carrier screeig through the Cousyl JScree research project, The Hexosamiidase A Variats of Ukow Sigificace (HAVUS). The goal of this project was to reclassify commo HEXA VUS to likely beig/beig, thereby coferrig a higher carrier detectio rate for sequecig. This research will be a meaigful milestoe i the evolutio for Tay-Sachs carrier screeig from ezyme aalysis to sequecig. The Aual Family Coferece is the corerstoe of NTSAD s Family Services program. Families impacted by all forms of Tay-Sachs, Sadhoff, GM1 ad Caava diseases come together to support ad empower each other through similar ad shared experieces. The program ecompasses all facets of livig with these diseases from peer support, research updates, care maagemet ad choices, to a focus o relatioships, siblig support ad how to be a advocate. 3 FULL DAYS OVER 50,000 $ IN HELPING HANDS GRANTS give to help families atted the 2017 Aual Family Coferece This project was fuded by NTSAD ad affiliate family foudatios. KNOW THE FACTS: 40+ Ashkeazi Jews, Frech-Caadias, ad Cajus... FAMILIES HELPED New Support Resources to 1 i 190 Irish-Americas... Care Tip Series for Families The first two videos of the series lauched i the summer of 2017 to educate families o issues relatig to carig for a child. The topics covered G-Tubes A to Z ad Motio & Massage. The series will expad i the summer of 2018 to iclude two more topics relatig to care. More tha 20 evets ad over a doze custom t-shirt campaigs Families rallied ad raised fuds for research from coast to coast icludig Caada 50% AFFECTED 2017 Fiscal Year udited Fiacial Statemets A Emma s Fud for Families This ew fud, started by the Artiia family i memory of their daughter, Emma, was established to allow affected families to apply for grats to use towards erichmet activities. It ca be used ay way a family feels would be beeficial for creatig their memories, such as trips to the zoo, photo shoots, tickets to a evet, a overight i a hotel with a pool, etc. Commuity Rallies aroud Family Over $101,000 raised, brigig the total to over $320,000 sice 2011 UNAFFECTED NTSAD ecourages meetig with a geetic couselor to uderstad your risks before gettig tested. of the geeral populatio... NTSADEvets 2017 Day of Hope: Families Drive Research... is a carrier of the Tay-Sachs gee. screeig Traslatig Patiet Experiece to Help Research NTSADFamily carrier NTSADResearch We would like to give a special thaks to the Roaldso family ad frieds for raisig over $50,000 for research with their Day of Hope evet. Mollie ad Madely, who have Juveile Sadhoff, are a extraordiary ispiratio to ot oly their fried ad frieds, but to families worldwide. SUPPORT & REVENUE EXPENSES $809,635 $711,679 31% Research NTSADAdvocacy NTSAD, with ispiratio ad guidace from the EveryLife Foudatio ad the Natioal Orgaizatio for Rare Disorders, cotiued to ecourage families to reach out to their represetatives throughout the year. Sharig their stories ca stress the importace of votig for legislatio that ca eable families to be whole ad healthy. IRemembrace Jaso Arbetter Seaby Clark Jeter DeJog Ava Demeshko Cristia Feradez Avi Forrester Cash Gray Kylie Hatchel Amelie Lewi Levi Lucero, Jr. Cora McDoald Jea McKizie Alyssa Mushi Decla O Coell Ia Ortega Rio Page Mackezie Plick Barro Posey Cooper Richards Josiah Scaparotti Talia Steckma Nikko Zollar Jauary 1, 2017 December 31, % Gifts 18% Coferece 13% Special Evets 9% Grats 8% Restricted Gifts 1% Other 37% Research 34% Family Services 12% Fudraisig 7% Educatio 7% Admiistratio 3% Advocacy

5 2017 Research Iitiative Grats Awarded NTSAD partered with Caava Research Illiois ad Cure GM1 Foudatio o Patiet Isights Networks (PIN) for Caava ad GM1 diseases. The PIN s purpose is to allow families to eter iformatio about their disease experiece that is traslated ito deidetified data to pait a picture of the disease experiece across a global commuity. The data will ultimately help promisig research advace to cliical trials by bolsterig the hard data of the research. Proof of cocept study of HSC gee therapy for Tay-Sachs disease NTSAD ad Cure Tay-Sachs Foudatio (CTSF) have partered to lauch a GM2 Tay-Sachs ad Sadhoff Disease Patiet Isights Network (PIN) this sprig. Millio Dollar Bike Rides Raise $40,000 for NTSAD Grat Team NTSAD raised $40,000 from the Fourth Aual Millio Dollar Bike Ride held i Philadelphia, ad the amout was matched by UPe s Orpha Disease Ceter! The 2017 grat wet to Igor Nestrasil, MD, PhD, at the Uiversity of Miesota for his project Brai MRI sigatures i ifats with ifatile forms of GM-1 ad GM-2. Pricipal Ivestigator: Alessadra Biffi, MD Daa-Farber/Bosto Childre s Cacer ad Blood Disorders Ceter Miimally ivasive delivery of AAV gee therapy i the Tay-Sachs Sheep Pricipal Ivestigator: Heather Gray-Edwards, PhD Scott-Ritchey Research Ceter, Aubur Uiversity, Aubur, AL Developmet of a quatitative method for the determiatio of a petasaccharide i GM1-gagliosidosis patiet cells to assess the potetial therapeutic efficacy of a beta-galactosidase pharmacological chaperoe drug cadidate. Pricipal Ivestigators: Tim Wood, PhD, Greewood Geetic Ceter, Greewood, South Carolia ad Stephae Demotz, PhD, Dorpha, Lausae, Switzerlad Accelerated program for CSF delivery of AAV gee therapy for Tay-Sachs ad Sadhoff patiets Pricipal Ivestigator: Miguel Sea-Esteves, PhD Uiversity of Massachusetts Medical School Progress i Gee Therapy Research The Tay-Sachs Gee Therapy Cosortium completed aimal studies ad the follow-up pathology studies. The results met the criteria to advace to the ext stage. They will ext maufacture cliical grade vectors with a goal i 2018 to submit a Ivestigatioal New Drug (IND) filig to start cliical trials. NTSADAwareess NTSAD s ANNUAL FAMILY CONFERENCE: A Commuity of Support Expaded Carrier Screeig Progress was made i carrier screeig through the Cousyl JScree research project, The Hexosamiidase A Variats of Ukow Sigificace (HAVUS). The goal of this project was to reclassify commo HEXA VUS to likely beig/beig, thereby coferrig a higher carrier detectio rate for sequecig. This research will be a meaigful milestoe i the evolutio for Tay-Sachs carrier screeig from ezyme aalysis to sequecig. The Aual Family Coferece is the corerstoe of NTSAD s Family Services program. Families impacted by all forms of Tay-Sachs, Sadhoff, GM1 ad Caava diseases come together to support ad empower each other through similar ad shared experieces. The program ecompasses all facets of livig with these diseases from peer support, research updates, care maagemet ad choices, to a focus o relatioships, siblig support ad how to be a advocate. 3 FULL DAYS OVER 50,000 $ IN HELPING HANDS GRANTS give to help families atted the 2017 Aual Family Coferece This project was fuded by NTSAD ad affiliate family foudatios. KNOW THE FACTS: 40+ Ashkeazi Jews, Frech-Caadias, ad Cajus... FAMILIES HELPED New Support Resources to 1 i 190 Irish-Americas... Care Tip Series for Families The first two videos of the series lauched i the summer of 2017 to educate families o issues relatig to carig for a child. The topics covered G-Tubes A to Z ad Motio & Massage. The series will expad i the summer of 2018 to iclude two more topics relatig to care. More tha 20 evets ad over a doze custom t-shirt campaigs Families rallied ad raised fuds for research from coast to coast icludig Caada 50% AFFECTED 2017 Fiscal Year udited Fiacial Statemets A Emma s Fud for Families This ew fud, started by the Artiia family i memory of their daughter, Emma, was established to allow affected families to apply for grats to use towards erichmet activities. It ca be used ay way a family feels would be beeficial for creatig their memories, such as trips to the zoo, photo shoots, tickets to a evet, a overight i a hotel with a pool, etc. Commuity Rallies aroud Family Over $101,000 raised, brigig the total to over $320,000 sice 2011 UNAFFECTED NTSAD ecourages meetig with a geetic couselor to uderstad your risks before gettig tested. of the geeral populatio... NTSADEvets 2017 Day of Hope: Families Drive Research... is a carrier of the Tay-Sachs gee. screeig Traslatig Patiet Experiece to Help Research NTSADFamily carrier NTSADResearch We would like to give a special thaks to the Roaldso family ad frieds for raisig over $50,000 for research with their Day of Hope evet. Mollie ad Madely, who have Juveile Sadhoff, are a extraordiary ispiratio to ot oly their fried ad frieds, but to families worldwide. SUPPORT & REVENUE EXPENSES $809,635 $711,679 31% Research NTSADAdvocacy NTSAD, with ispiratio ad guidace from the EveryLife Foudatio ad the Natioal Orgaizatio for Rare Disorders, cotiued to ecourage families to reach out to their represetatives throughout the year. Sharig their stories ca stress the importace of votig for legislatio that ca eable families to be whole ad healthy. IRemembrace Jaso Arbetter Seaby Clark Jeter DeJog Ava Demeshko Cristia Feradez Avi Forrester Cash Gray Kylie Hatchel Amelie Lewi Levi Lucero, Jr. Cora McDoald Jea McKizie Alyssa Mushi Decla O Coell Ia Ortega Rio Page Mackezie Plick Barro Posey Cooper Richards Josiah Scaparotti Talia Steckma Nikko Zollar Jauary 1, 2017 December 31, % Gifts 18% Coferece 13% Special Evets 9% Grats 8% Restricted Gifts 1% Other 37% Research 34% Family Services 12% Fudraisig 7% Educatio 7% Admiistratio 3% Advocacy

6 NTSAD Spotlight o Imagie & Believe NTSAD recogized its 60th aiversary throughout 2017 by hoorig those families ad idividuals who established ad expaded the NTSAD commuity, by recogizig our preset achievemets, ad by embracig goals for the future i the areas of family support, research advaces, ad prevetative outreach NTSAD celebrated its 60th Aiversary Imagie & Believe evet at the Royal Soesta i Cambridge o Thursday, November 9th. Highlights of the ight icluded a thoughtful discussio with hoorees Dr. David Meeker ad Professor Timothy Cox. Moderated by NTSAD s former Executive Director, Jaye Gershkowitz, topics icluded what motivates their work i the rare disease commuity, scietific progressios, ad the value that a orgaizatio like NTSAD ca have o both families ad supporters. This discussio was followed by heartfelt remarks by Talia s mother ad NTSAD paret, Carla Steckma. Joiig Carla ad hoorees, David ad Tim, were 190 guests who raised over $120,000 the evet s largest total sice its iceptio! BOARD OF DIRECTORS SCIENTIFIC ADVISORY COMMITTEE Presidet Blyth Lord Chairs Fraces Platt, PhD Jodi Hoffma, MD (Vice Chair) Vice Presidet Staci Kallish, DO NTSADMissio NTSAD leads the fight to treat ad cure Tay-Sachs, Sadhoff, GM1, Caava ad related geetic diseases, ad supports affected families ad idividuals i leadig fuller lives. Meryl Kallish Staci Kallish Eda Kapla Amy Katz, Esq. 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 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 Michael Richards Corporate Advisory Coucil Oved Amitay Ritu Baral Natioal Tay-Sachs & Allied Diseases Associatio Laura Cohe Joh Gordo Michael Gladstoe Kate Havilad Walt Kowtoiuk, PhD Greg Licholai, MD Joa Keutzer, PhD 2001 Beaco Street Suite 204 Bosto, Massachusetts (617) (800) 90-NTSAD Treasurer Alex Wright Secretary Ruth Feldma Directors Merle Adelma Stewart Altma Risa Ase Moica Gettlema Kare Grizaid Oralea Marquardt Alec Rubestei Amy Speak Mario Yaovsky Past Presidets Bria Maig Thomas P. Lord Joh F. Crowley, JD Mark P. Madse Bradley Campbell Natioal Tay-Sachs & Allied Diseases Associatio Executive Director Susa R. Kah Director of Developmet Patrick Woods Director of Family Services Diaa Pagois Yvoe Sacks Developmet & Admiistrative Associate Tsitsi Moyo Sedra Schiffma Coferece Coordiator Becky Beso Jaye Mackta Judith Saperstei Claire Kah Evely Sussma* Ruth Dukell* * deceased ad educatio. Desig by Beth Bedavid-Val 2017 I ANNUAL REPORT