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2014 Annual Meeting | Muscle Disease: Gene Therapy and Models

Thursday 05/01/14
03:15 PM - 05:00 PM Add To Calendar
This program will be presented both in-person and online
This program is not expected to be available in the meeting's On Demand product.
Scientific Platform Session
Neuromuscular and Clinical Neurophysiology (EMG)
No CME available
Event Timeline
03:15 PM - 05:00 PM Abstract 001 - Restoration of dystrophin expression in a novel Duchenne mouse model, the Dup2 mouse, by IRES-mediated translation of a highly functional N-truncated dystrophin protein
Kevin M. Flanigan, MD, FAAN
03:15 PM - 05:00 PM Abstract 002 - ?-Sarcoglycan Gene Transfer Leads to Functional Improvement in a Model of LGMD2E
Eric Pozsgai
03:15 PM - 05:00 PM Abstract 003 - MicroRNA-29 overexpression delivered by adeno-associated virus suppresses fibrosis in mdx:utrn+/- mice
Eric Meadows
03:15 PM - 05:00 PM Abstract 004 - AAV Gene Transfer Utilizing Homologous Overlap Vectors Mediates Functional Recovery of Dysferlin Deficiency
Patricia C. Sondergaard
03:15 PM - 05:00 PM Abstract 005 - Fhl1 W122S knock-in mice manifest late-onset mild myopathy
Valentina Emmanuele, MD, PhD
03:15 PM - 05:00 PM Abstract 006 - Impaired Muscle Regeneration in CAPN3-KO Mice related to microRNA dysregulation
Zarife Sahenk, MD, PhD, FAAN
03:15 PM - 05:00 PM Abstract 007 - Mutations in zaspopathy cause disruption of actinin-crosslinked actin filaments, the core structure of skeletal muscle Z-discs
Xiaoyan Lin
Faculty Disclosures
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 MT Pharma. 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 $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. 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.
Nicholas E. Johnson, MD, FAAN Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta Therapuetics. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne Therapeutics. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharma. Dr. Johnson has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for ML BIo . Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity. Dr. Johnson has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Arthex. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Juvena. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Entrada. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Rgenta. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Angle Therapeutics. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AskBio. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum. Dr. Johnson has stock in Angle Therapeutics. Dr. Johnson has stock in Juvena Therapeutics. The institution of Dr. Johnson has received research support from Novartis. The institution of Dr. Johnson has received research support from Takeda . The institution of Dr. Johnson has received research support from AMO Pharma. The institution of Dr. Johnson has received research support from Sarepta. The institution of Dr. Johnson has received research support from Dyne Therapeutics. The institution of Dr. Johnson has received research support from AskBio. The institution of Dr. Johnson has received research support from Pepgen. The institution of Dr. Johnson has received research support from ML Bio. The institution of Dr. Johnson has received research support from Vertex. The institution of Dr. Johnson has received research support from Arthex. Dr. Johnson has received intellectual property interests from a discovery or technology relating to health care.
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.
Michio Hirano, MD, FAAN Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Modis Therapeutics (a subsidiary of Zogenix). Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epirium Bio. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Innovation Specialist. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Precision Biosciences. Dr. Hirano has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Platform Q Health. Dr. Hirano has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Stealth Biotherapeutics. The institution of Dr. Hirano has received research support from Modis Therapeutics (a subsidiary of Zogenix. The institution of Dr. Hirano has received research support from Cyclerion. Dr. Hirano has received research support from Reneo Pharmaceuticals. The institution of Dr. Hirano has received research support from Astellas. Dr. Hirano has received intellectual property interests from a discovery or technology relating to health care. Dr. Hirano has received intellectual property interests from a discovery or technology relating to health care. Dr. Hirano has received personal compensation in the range of $0-$499 for serving as a Study Section Reviewer with NIH. Dr. Hirano has a non-compensated relationship as a Research Advisory Board member with Muscular Dystrophy Association that is relevant to AAN interests or activities. Dr. Hirano has a non-compensated relationship as a Scientific and Medical Advisory Board member with United Mitochondrial Disease Foundation that is relevant to AAN interests or activities. Dr. Hirano has a non-compensated relationship as a Scientific Advisory Board member with Barth Syndrome Foundation that is relevant to AAN interests or activities.
Kevin M. Flanigan, MD, FAAN Dr. Flanigan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Apic Bio. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AavantiBio. Dr. Flanigan has stock in 4D Molecular Therapeutics. The institution of Dr. Flanigan has received research support from Abeona Therapeutics. The institution of Dr. Flanigan has received research support from Sarepta Therapeutics. The institution of Dr. Flanigan has received research support from Astellas Therapeutics. Dr. Flanigan has received intellectual property interests from a discovery or technology relating to health care.
K. Reed Clark, PhD No disclosure on file
Robert C. Griggs, MD, FAAN Dr. Griggs has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Strongbridge Pharmaceuticals. Dr. Griggs has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Idera Pharmaceuticals. Dr. Griggs has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Biopharma. Dr. Griggs has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Santhera Pharmaceuticals. Dr. Griggs 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. Griggs has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Lippincott. The institution of Dr. Griggs has received research support from PTC Pharmaceuticals. The institution of Dr. Griggs has received research support from Sarepta Pharmaceuticals. The institution of Dr. Griggs has received research support from National Institutes of Health. The institution of Dr. Griggs has received research support from Santhere Pharmaceuticals. Dr. Griggs has received personal compensation in the range of $500-$4,999 for serving as a Study section member with National Institutes of Health. Dr. Griggs has a non-compensated relationship as a Board of Directors;Chair Research Advisory Committee with American Brain Foundation that is relevant to AAN interests or activities.
No disclosure on file
Valentina Emmanuele, MD, PhD No disclosure on file
Beatriz Garcia-Diaz No disclosure on file
Eric Meadows No disclosure on file
Caterina Garone No disclosure on file
Lei Chen No disclosure on file
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.
Patricia C. Sondergaard No disclosure on file
Tomas Kalincik Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi Genzyme. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Merck. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Tomas Kalincik has received personal compensation in the range of $500-$4,999 for serving as a Consultant for National Health and Medical Research Council. Tomas Kalincik has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MS Research Australia. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Tomas Kalincik 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. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Genzyme. Tomas Kalincik 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. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Tomas Kalincik has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Tomas Kalincik has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bristol Myers Squibb. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for WebMD Global. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Novartis. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Sanofi Genzyme. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Teva. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for BioCSL. Tomas Kalincik has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Merck. Tomas Kalincik has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Eisai. The institution of Tomas Kalincik has received research support from Biogen. The institution of Tomas Kalincik has received research support from Novartis. The institution of Tomas Kalincik has received research support from Genzyme. The institution of Tomas Kalincik has received research support from Roche. The institution of Tomas Kalincik has received research support from Celgene. The institution of Tomas Kalincik has received research support from Merck. The institution of Tomas Kalincik has received research support from MSBase Foundation. The institution of Tomas Kalincik has received research support from ARSEP and EDMUS Foundations. The institution of Tomas Kalincik has received research support from MS Research Australia. The institution of Tomas Kalincik has received research support from National Health and Medical Research Council. The institution of Tomas Kalincik has received research support from Australian Research Council. The institution of Tomas Kalincik has received research support from MS Society. The institution of Tomas Kalincik has received research support from Trish Foundation. Tomas Kalincik has a non-compensated relationship as a scientific leadership group vice-chair with MSBase Foundation that is relevant to AAN interests or activities.
Zarife Sahenk, MD, PhD, FAAN Dr. Sahenk 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.
Kenneth H. Fischbeck, MD, FAAN Dr. Fischbeck has received research support from NINDS/NIH. Dr. Fischbeck has received intellectual property interests from a discovery or technology relating to health care. Dr. Fischbeck has a non-compensated relationship as a Scientific Review Board member with Kennedy's Disease Association that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a Scientific Council member with Association Francaise contre les Myopathies that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a Independent Review Committee member with Target ALS that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a Scientific Advisory Board member, Musculoskeletal Diseases with Novartis that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a Senior Clinical Consultant with n-Lorem Foundation that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a Scientific Advisory Committee member with Stanford GNE myopathy program that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a Scientific Advisory Board member with Hereditary Disease Foundation that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a Scientific Advisory Board member with Packard Center for ALS Research that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a Scientific Advisory Board member with Huntington's Disease Society of America that is relevant to AAN interests or activities. Dr. Fischbeck has a non-compensated relationship as a TACT review panel member with TREAT-NMD that is relevant to AAN interests or activities.
Catarina Quinzii Hirano, MD No disclosure on file
Sarah E. Lewis Mrs. Lewis has received personal compensation for serving as an employee of Sarepta Therapeutics . Mrs. Lewis has stock in Sarepta Therapeutics.
Orhan H. Akman, PhD No disclosure on file
Ami K. Mankodi, MD, FAAN No disclosure on file
Rachel Ohman No disclosure on file
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.
Kristin N. Heller, PhD No disclosure on file
Reed Clark No disclosure on file
Janaiah Kota No disclosure on file
Xiaoyan Lin No disclosure on file
Vinod Malik No disclosure on file
Henry J. Kaminski, MD, FAAN The institution of Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Kaminski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio. Dr. Kaminski has stock in ARC Biotechnology. The institution of Dr. Kaminski has received research support from National Institutes of Health. Dr. Kaminski has received publishing royalties from a publication relating to health care.
Wai-Kuen Leong, MD Dr. Leong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen Australia.
Kevin O'Connor, FRACP, MB, BCh, BAO No disclosure on file