2021 Annual Meeting | S32 - Therapeutics of Genetic Neuromuscular Diseases
Program Materials Program Evaluations
| 04:00 PM - 04:08 PM EDT | Abstract |
001 - Global Open-label Extension: 24-month Data in Patients with hATTR Amyloidosis
Michael J. Polydefkis, MD, FAAN |
| 04:08 PM - 04:16 PM EDT | Abstract |
002 - Impact of Patisiran on Activities of Daily Living and Functional Status in hATTR Amyloidosis
Amanda C. Peltier, MD, MS |
| 04:16 PM - 04:24 PM EDT | Q&A |
Questions and Answers
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| 04:24 PM - 04:32 PM EDT | Abstract |
003 - Safety, ß-sarcoglycan Expression and Functional Outcomes from Systemic Gene Transfer of rAAVrh74.MHCK7.SGCB in Patients with Limb-Girdle Muscular Dystrophy Type 2E (LGMD2E)
Louise R. Rodino-Klapac |
| 04:32 PM - 04:40 PM EDT | Abstract |
004 - A Multicenter Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular Dystrophy
Jerry R. Mendell, MD, FAAN |
| 04:40 PM - 04:48 PM EDT | Abstract |
005 - AAV gene therapy for TNNT1-associated Nemaline myopathy
Eleonora D'Ambrosio, MD |
| 04:48 PM - 05:00 PM EDT | Q&A |
Questions and Answers
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| Zarife Sahenk, MD, PhD, FAAN | Dr. Sahenk has nothing to disclose. |
| Madeline Merkel | Madeline Merkel has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. |
| No disclosure on file | |
| Amanda C. Peltier, MD, MS | Dr. Peltier has received personal compensation for serving as an employee of Alnylam. Dr. Peltier has received personal compensation for serving as an employee of Akcea. Dr. Peltier has received personal compensation for serving as an employee of CSL Behring. Dr. Peltier has received personal compensation for serving as an employee of Catalyst. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Csl Behring. Dr. Peltier has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Peltier has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Akcea. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for CSL Behring. Dr. Peltier has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for catalyst. The institution of Dr. Peltier has received research support from NIH. |
| Rachael Potter | No disclosure on file |
| Tejdip Singh | Tejdip Singh has received personal compensation for serving as an employee of Sarepta Therapeutics . Tejdip Singh has received stock or an ownership interest from Sarepta Therapeutics. |
| Senda Ajroud-Driss, MD, FAAN | Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx Pharmaceuticals. Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Ajroud-Driss has received research support from ALSA. Dr. Ajroud-Driss has received publishing royalties from a publication relating to health care. |
| Ole Suhr | The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam . The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Prothena. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Intellia. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akces. The institution of Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alnylam. Ole Suhr has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Akcea. The institution of Ole Suhr has received research support from Swedish Heart and Lung Foundation. |
| Kevin Strauss | Kevin Strauss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AveXis. |
| Jing Jing Wang, MD | No disclosure on file |
| Perry Shieh, MD, PhD, FAAN | Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for CSL Behring. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Grifols. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Shieh has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Biogen. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Genentech. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Catalyst. |
| Kathleen Church | No disclosure on file |
| Lindsay N. Alfano, PT | Ms. Alfano has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ATOM International, Ltd (Amicus Therapeutics, Catabasis, Genethon, Italfarmaco, NS Pharma, Pfizer, PTC Therapeutics). Ms. Alfano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Novartis Gene Therapies. The institution of Ms. Alfano has received research support from Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Audentes Therapeutics/Astellas Gene Therapies. Ms. Alfano has received intellectual property interests from a discovery or technology relating to health care. |
| Megan Iammarino, PT | Dr. Iammarino has nothing to disclose. |
| Kelly Lehman, MSN, CNP | Ms. Lehman has nothing to disclose. |
| Linda P. Lowes, PT PhD | The institution of Ms. Lowes has received research support from Sarepta Therapeutics. |
| Elizabeth A. Mauricio, MD, FAAN | Dr. Mauricio has nothing to disclose. |
| Teresa Coelho, MD | Dr. Coelho has nothing to disclose. |
| John L. Berk | John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca/IONIS. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eidos/BridgBio. John L. Berk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Intellia Therapeutics. John L. Berk has received research support from Alnylam . John L. Berk has received research support from Ionis. John L. Berk has received research support from Eidos/Bridgbio. |
| Louise R. Rodino-Klapac | Ms. Rodino-Klapac has received personal compensation for serving as an employee of Sarepta Therapeutics, Inc.. Ms. Rodino-Klapac has received stock or an ownership interest from Sarepta Therapeutics. Ms. Rodino-Klapac has received intellectual property interests from a discovery or technology relating to health care. |
| Danielle A. Griffin | Ms. Griffin has stock in Sarepta Therapeutics. Ms. Griffin has received intellectual property interests from a discovery or technology relating to health care. |
| Eric Pozsgai | Eric Pozsgai has received personal compensation for serving as an employee of Sarepta Therapeutics. Eric Pozsgai has received stock or an ownership interest from Sarepta Therapeutics. |
| Marianne T. Sweetser, MD, PhD | Dr. Sweetser has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Dr. Sweetser has received stock or an ownership interest from Alnylam Pharmaceuticals. |
| Thomas Brannagan III, MD, FAAN | Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Grifols. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alnylam. The institution of Dr. Brannagan has received research support from Alnylam. The institution of Dr. Brannagan has received research support from Acceleron. The institution of Dr. Brannagan has received research support from Ionis. The institution of Dr. Brannagan has received research support from Pharnext. |
| David D. Adams | David D. Adams has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ALNYLAM. David D. Adams has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. |
| Jerry R. Mendell, MD, FAAN | Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vertex. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics . The institution of Dr. Mendell has received research support from Sarepta. |
| Michael J. Polydefkis, MD, FAAN | Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharmaceutical . Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen-Idec. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. |
| Natalie F. Miller, PT | Dr. Miller has received personal compensation for serving as an employee of ATOM International. Dr. Miller has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Casimir. |
| Eleonora D'Ambrosio, MD | Dr. D'Ambrosio has nothing to disclose. |
| Srikanth Muppidi, MD, FAAN | Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizont Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care. |
| Lauren B. Elman, MD | Dr. Elman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Elman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche/Genentech. Dr. Elman has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Edgewise Therapeutics. Dr. Elman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Dr. Elman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Apellis Pharamaceuticals. Dr. Elman has received publishing royalties from a publication relating to health care. |