2017 Annual Meeting | S27 - "Best Of" Session: Neuromuscular Disease Therapeutics
| 08:00 AM - 09:00 AM | Abstract |
001 - Rasagiline for the Treatment of ALS: a randomized controlled study
Richard J. Barohn, MD, FAAN |
| 08:00 AM - 09:00 AM | Abstract |
002 - Oral Fingolimod in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (FORCIDP): Results from a Phase III Randomized Placebo-controlled Trial
Hans-Peter Hartung, MD, FAAN |
| 08:00 AM - 09:00 AM | Abstract |
003 - AVXS-101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Interim Data Demonstrates Improvements in Supportive Care Use
Jerry R. Mendell, MD, FAAN |
| 08:00 AM - 09:00 AM | Abstract |
004 - Phase 2 Open-Label Extension (OLE) Study of Patisiran, an Investigational RNA interference (RNAi) Therapeutic for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy
David D. Adams |
| 08:35 AM - 08:45 AM | Speaker |
Panel Discussion
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| 08:45 AM - 09:00 AM | Speaker |
Meet the Investigators
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| Tahseen Mozaffar, MD, FAAN | Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas Gene Therapy. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics. The institution of Dr. Mozaffar has received research support from NIH. The institution of Dr. Mozaffar has received research support from Muscular Dystrophy Association. The institution of Dr. Mozaffar has received research support from Sanofi. The institution of Dr. Mozaffar has received research support from Argenx. The institution of Dr. Mozaffar has received research support from Amicus Therapeutics. The institution of Dr. Mozaffar has received research support from Astellas Gene Therpay. The institution of Dr. Mozaffar has received research support from Cartesian. The institution of Dr. Mozaffar has received research support from Cabaletta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH. |
| Suur Biliciler, MD | Dr. Biliciler has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Efficient CME. Dr. Biliciler has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenyx. Dr. Biliciler has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for UCB. |
| No disclosure on file | |
| William Martens | William Martens has nothing to disclose. |
| Nuran Dilek | Nuran Dilek has nothing to disclose. |
| Jeffrey Statland, MD | Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vita Therapeutics. Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex . The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA. |
| Ghazala Hayat, MBBS, FAAN | Dr. Hayat has received personal compensation in the range of $500-$4,999 for serving as a Consultant for kabafusion. Dr. Hayat 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. Dr. Hayat has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for alexion. Dr. Hayat has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for MTPA. Dr. Hayat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for alexion. Dr. Hayat has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for kabafusion. |
| Linda P. Lowes, PT PhD | The institution of Ms. Lowes has received research support from Sarepta Therapeutics. |
| Richard A. Hughes, MD | Dr. Hughes has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. |
| Jean-Marc Leger, MD, FAAN | No disclosure on file |
| Katherine L. Berry, MD | No disclosure on file |
| Chafic Y. Karam, MD | Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Karam has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neuroderm. |
| Marcia Waddington Cruz, MD | Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sobi. Dr. Waddington Cruz 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. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ionis. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NovoNordisk. |
| Isabel Conceicao | Isabel Conceicao has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Isabel Conceicao has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sobi. |
| Gen Sobue, MD | Dr. Sobue has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Mistubishi Tanabe Pharma Corporation. Dr. Sobue has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Nippon Chemiphar Co., Ltd.. Dr. Sobue has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Mistubishi Tanabe Pharma Corporation. Dr. Sobue has received personal compensation in the range of $0-$499 for serving as an Expert Witness for Takeda Pharmaceutical Company Limited.. |
| Jean Pouget, MD, PhD | No disclosure on file |
| Sukumar Nagendran, MD | No disclosure on file |
| 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. |
| Samiah A. Al-Zaidy, MD | Dr. Al-Zaidy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AveXis Inc. |
| Marinos C. Dalakas, MD, FAAN | Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols, . Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx,. Dr. Dalakas has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Dysimmune Diseases Foundation. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Serono. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octapharma. Dr. Dalakas has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Kyverna. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ARGENX. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for AAN. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Therapeutic Advances in Neurology (TAND). Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Medlink. |
| Kathleen Church | No disclosure on file |
| Arthur H. Burghes, MD | No disclosure on file |
| Ingemar S. Merkies, MD | No disclosure on file |
| 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. |
| Brian K. Kaspar, PhD | No disclosure on file |
| Minna Du | No disclosure on file |
| Douglas M. Sproule, MD | Dr. Sproule has received personal compensation for serving as an employee of Bridge Bio. Dr. Sproule has received personal compensation for serving as an employee of Novartis, Inc. Dr. Sproule has received stock or an ownership interest from Bridge Bio. |
| Norman Latov, MD, PhD | Dr. Latov has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Polyneuron. Dr. Latov has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Therapath LLC. Dr. Latov has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Mcaloon & Freeman, PC. Dr. Latov has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Conway Homer PC. Dr. Latov has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Sylvia Chin-Kaplan, LLC. The institution of Dr. Latov has received research support from Takeda. Dr. Latov has received publishing royalties from a publication relating to health care. |
| Teresa Coelho, MD | Dr. Coelho has nothing to disclose. |
| 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. |
| 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. |
| John T. Kissel, MD, FAAN | Dr. Kissel has nothing to disclose. |
| Hans-Peter Hartung, MD, FAAN | Dr. Hartung has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BMS Celgene. Dr. Hartung has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Geneuro. Dr. Hartung has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Hartung has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Hartung has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Dr. Hartung has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bayer. Dr. Hartung has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TG Therapeutics. Dr. Hartung has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Hartung has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Frontiers Neurology. |
| Martin Bernhard Merschhemke | Martin Bernhard Merschhemke has received personal compensation for serving as an employee of Novartis Pharma AG. Martin Bernhard Merschhemke has received stock or an ownership interest from Novartis Pharma AG. |
| Dan Moore, PhD | No disclosure on file |
| J. Americo M. Fernandes, Jr., MD, FAAN | Dr. Fernandes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Columbia University. The institution of Dr. Fernandes has received research support from MGH philanthropy (Clene, Seelos, UCB, Biohaven, Prilenia, Denali Therapeutics, Calico Life Sciences. The institution of Dr. Fernandes has received research support from Columbia University. The institution of Dr. Fernandes has received research support from PTC Therapeutics. The institution of Dr. Fernandes has received research support from Clene. |
| 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. |
| Hiroshi Mitsumoto, MD, FAAN | Dr. Mitsumoto has nothing to disclose. |
| Sharon P. Nations, MD, FAAN | No disclosure on file |
| Laura Herbelin | No disclosure on file |
| Jonathan S. Katz, MD | Dr. Katz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Katz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe. Dr. Katz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Calico. Dr. Katz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL Behring. Dr. Katz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenyx. Dr. Katz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Katz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Denali. Dr. Katz has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Griffols. Dr. Katz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Katz has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. |
| David S. Saperstein, MD | No disclosure on file |
| Angela L. Genge, MD | Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AL-S Pharma. Dr. Genge has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amylyx. Dr. Genge has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Quralis. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Genge has received personal compensation in the range of $0-$499 for serving as a Consultant for WAVE. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for eikonizo. Dr. Genge has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for pepgen. Dr. Genge has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for rapa. |
| 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. |
| William D. Arnold, MD | Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for La Hoffmann Roche. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cadent Therapeutics . Dr. Arnold has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. The institution of Dr. Arnold has received research support from NIH. The institution of Dr. Arnold has received research support from NMD Pharma. The institution of Dr. Arnold has received research support from Gilead Sciences. The institution of Dr. Arnold has received research support from CureSMA. Dr. Arnold has received intellectual property interests from a discovery or technology relating to health care. |
| 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. |
| Eduardo Nobile-Orazio, MD, PhD, FAAN | Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ArgenX, Belgium. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for LFB, France. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche, Switzerland. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL Behring. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen, USA. The institution of Dr. Nobile-Orazio has received research support from Takeda/Shire. |
| 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. |
| Jared Gollob, MD | No disclosure on file |
| Richard J. Barohn, MD, FAAN | Dr. Barohn has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for NuFactor. The institution of Dr. Barohn has received research support from FDA OPD R01. Dr. Barohn has received intellectual property interests from a discovery or technology relating to health care. |