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2010 Annual Meeting | Emerging Treatments in Neuromuscular Disease

Wednesday 04/14/10
02:00 PM - 06:00 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.
Integrated Neuroscience
Muscle Disease/Neuromuscular Junction
No CME available
Event Timeline
02:00 PM - 06:00 PM Abstract 001 - Preliminary Results with AVI-4658 of Dystrophin Expression, Safety and Pharmacokinetics from the First Systemic Administration Study in Boys with Duchene Muscular Dystrophy (DMD), with a Phosphorodiamidate Morpholino Oligomer (PMO) to Skip Exon 51
02:00 PM - 06:00 PM Abstract 001 - Single, High Doses of Epoetin alfa Increase Frataxin in Patients with Friedreich[apos]s Ataxia
02:00 PM - 06:00 PM Abstract 001 - Complete Rescue of SMA Mouse Model by Systemic Delivery of scAAV9 Mediating SMN Replacement
02:00 PM - 06:00 PM Abstract 002 - A Phase I/IIa Systemic Study on Antisense Oligonucleotide Compound PRO051 in Patients with Duchenne Muscular Dystrophy
02:00 PM - 06:00 PM Abstract 002 - Intrathecal Delivery of Autologous Mesenchymal Stem Cells in Patients with Amyotrophic Lateral Sclerosis
02:00 PM - 06:00 PM Abstract 002 - Antisense Therapeutics for the Treatment of Neurodegenerative Disorders: Preclinical Studies
02:00 PM - 06:00 PM Abstract 003 - Clinical Trial Testing Lithium Carbonate and Riluzole in Patients with ALS Terminates Early for Futility
02:00 PM - 06:00 PM Abstract 003 - Prolonged Administration of High Dose Ascorbic Acid Impairs Rat Sciatic Nerve Functional Recovery after Crush Injury
02:00 PM - 06:00 PM Abstract 003 - Pharmacokinetics of IV Immunoglobulin and Outcome in Guillain-Barr[eacute] Syndrome
02:00 PM - 06:00 PM Abstract 004 - ACE-031 Significantly Increases Lean Body Mass and Muscle Volume in Healthy Postmenopausal Volunteers Following a Single Subcutaneous Dose
02:00 PM - 06:00 PM Abstract 004 - Pulsed Electromagnetic Fields To Reduce Diabetic Neuropathic Pain and Stimulate Neuronal Repair: A Randomized Controlled Trial
02:00 PM - 06:00 PM Abstract 005 - Adult Murine Derived Mesoangioblasts Successfully Recovered Dysferlin Expression in a Murine Model of Dysferlinopathy
02:00 PM - 06:00 PM Abstract 005 - Tafamidis (Fx-1006A): A First-In-Class Disease-Modifying Therapy for Transthyretin Type Familial Amyloid Polyneuropathy
02:00 PM - 06:00 PM Abstract 006 - Initial Efficacy and Safety Evaluation in Cynomolgus Monkeys of AVI-5038, a Peptide Conjugated Phosphorodiamidate Morpholino Oligomer (PPMO) Being Developed To Skip Exon 50 in Duchenne Muscular Dystrophy
02:00 PM - 06:00 PM Abstract 007 - Proof-of-Concept for a Novel Small Molecule Hsp90 Inhibitor for Spinal and Bulbar Muscular Atrophy (SBMA)
Faculty Disclosures
Misha-Miroslav Backonja, MD Dr. Backonja 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.
Allan E. Rubenstein, MD No disclosure on file
Richard A. Smith, MD Dr. Smith has received personal compensation for serving as an employee of Center for Neurologic Study. Dr. Smith has received intellectual property interests from a discovery or technology relating to health care. Dr. Smith has received intellectual property interests from a discovery or technology relating to health care. Dr. Smith has received publishing royalties from a publication relating to health care. Dr. Smith has received personal compensation in the range of $10,000-$49,999 for serving as a Inventor with Ionis Pharmaceutical.
Charles A. Thornton, MD, FAAN Dr. Thornton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity. Dr. Thornton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Thornton has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Ionis. Dr. Thornton has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne. Dr. Thornton has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pepgen. The institution of Dr. Thornton has received research support from Marigold Foundation. The institution of Dr. Thornton has received research support from NIH. The institution of Dr. Thornton has received research support from Muscular Dystrophy Association . The institution of Dr. Thornton has received research support from Dyne. The institution of Dr. Thornton has received research support from Avidity. The institution of Dr. Thornton has received research support from Ionis. Dr. Thornton has received intellectual property interests from a discovery or technology relating to health care.
Michael I. Weintraub, MD, FACP, PC, FAAN Dr. Weintraub has nothing to disclose.
Robin A. Conwit, MD, FAAN Dr. Conwit has nothing to disclose.
Itzik Wirguin, MD No disclosure on file
Isabel Illa, MD No disclosure on file
Alessandro Filla, MD, FAAN No disclosure on file
A. Gordon Smith, MD, FAAN Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merz. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sangamo. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for US Department of Justice. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Smith has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Eidos. Dr. Smith has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Lexicon. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. The institution of Dr. Smith has received research support from NIH (NINDS, NIDDK). Dr. Smith has received publishing royalties from a publication relating to health care. Dr. Smith has received personal compensation in the range of $0-$499 for serving as a Study Section with NIH.
Nicholas J. Maragakis, MD, FAAN Dr. Maragakis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UptoDate. Dr. Maragakis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Apellis Pharmaceuticals. Dr. Maragakis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. An immediate family member of Dr. Maragakis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for National Basketball Association. Dr. Maragakis has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Akava. An immediate family member of Dr. Maragakis has received personal compensation in the range of $0-$499 for serving as an officer or member of the Board of Directors for Johns Hopkins Howard County Medical Center. Dr. Maragakis has stock in Akava. The institution of Dr. Maragakis has received research support from NIH/NINDS. The institution of Dr. Maragakis has received research support from Department of Defense. The institution of Dr. Maragakis has received research support from Maryland Stem Cell Research Fund. Dr. Maragakis has received intellectual property interests from a discovery or technology relating to health care.
Violaine Plante-Bordeneuve, MD Dr. Plante-Bordeneuve has nothing to disclose.
David N. Herrmann, MD, FAAN Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Acceleron. Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint global. Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GLG. Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurogene. Dr. Herrmann has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Herrmann has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regenacy. Dr. Herrmann has received intellectual property interests from a discovery or technology relating to health care.
Giuseppe De Michele, MD No disclosure on file
Alireza Minagar, MD No disclosure on file
Eric Haltom No disclosure on file
Eduard Gallardo No disclosure on file
Clementine Karageorgiou, MD The institution of Dr. Karageorgiou has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for GENESIS PHARMA. The institution of Dr. Karageorgiou has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for TEVA-SPECIFAR.
Yvan Torrente, MD No disclosure on file
Lorne H. Zinman, MD Dr. Zinman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen, MTP, AB Science, Cytokinetics, Amylyx. Dr. Zinman has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx.
Don Cleveland, PhD No disclosure on file
Kate Bushby, MD Dr. Bushby has nothing to disclose.
Elissaios Karageorgiou, MD, PhD, FAAN Dr. Karageorgiou has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Myrtelle. The institution of Dr. Karageorgiou has received research support from European Union.
Francesco Muntoni, MD Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Muntoni 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. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Biogen. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. The institution of Dr. Muntoni has received research support from European Commission. The institution of Dr. Muntoni has received research support from Medical Research Council. The institution of Dr. Muntoni has received research support from Biogen. The institution of Dr. Muntoni has received research support from Muscular Dystrophy UK. The institution of Dr. Muntoni has received research support from MDA USA. The institution of Dr. Muntoni has received research support from Sarepta. The institution of Dr. Muntoni has received research support from Association Francoise Myopathies. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving as a Clinical expert with UK NICE Committee.
Baki Goksan, PhD No disclosure on file
Bart C. Jacobs No disclosure on file
Swati Aggarwal, MD No disclosure on file
Gunnar Buyse, MD, PhD No disclosure on file
Stephen B. Shrewsbury, MD Dr. Shrewsbury has received personal compensation for serving as an employee of Impel NeuroPharma. Dr. Shrewsbury has received stock or an ownership interest from Impel NeuroPharma.
No disclosure on file
Francesco Sacca, MD Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lexeo. Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genpharm. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medpharma. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Madison Pharma. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zai Lab. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Reata. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sandoz. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neopharm Israel. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson&Johnson. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. The institution of Dr. Sacca has received research support from AIFA. The institution of Dr. Sacca has received research support from FARA.
Nathalie M. Goemans, MD Dr. Goemans 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. Goemans has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Dr. Goemans has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Dr. Goemans has received publishing royalties from a publication relating to health care.
Pieter Van Doorn, MD The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hansa. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Octapharma. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Anexxon. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Grifols. Dr. Van Doorn has received publishing royalties from a publication relating to health care.
Michela Guglieri The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne. The institution of Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NS Pharma. Michela Guglieri has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. The institution of Michela Guglieri has received research support from EC - H2020. The institution of Michela Guglieri has received research support from Duchenne UK. The institution of Michela Guglieri has received research support from Sarepta. The institution of Michela Guglieri has received research support from DMD Research Fund.
Pierre Lozeron No disclosure on file
Eleni Papageorgiou No disclosure on file
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.
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.
Niels Borgstein, MD No disclosure on file
Giorgos Tagaris No disclosure on file
Teresa Coelho, MD Dr. Coelho has nothing to disclose.
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.
Donna Grogan, MD No disclosure on file
Luis F. Maia No disclosure on file
Ana Martins Da Silva No disclosure on file
Jasbir Seehra, PhD No disclosure on file
Clotilde Balucani, MD, PhD Dr. Balucani has nothing to disclose.
Athanasia Alexoudi, MD No disclosure on file
Ioanna Chatzi No disclosure on file
Krista Kuitwaard, MD No disclosure on file
Alice Favaretto No disclosure on file
Richard S. Geary No disclosure on file
Holly Kordasiewicz Holly Kordasiewcz has received personal compensation for serving as an employee of Ionis. Holly Kordasiewcz has stock in Ionis.
Ke Ning, MD, PhD No disclosure on file
Francesca Rinaldi No disclosure on file
Zahara M. Jaffer, PhD No disclosure on file
Matthew L. Sherman, MD No disclosure on file
Peter Sazani, PhD No disclosure on file
Chiara Valori, PhD No disclosure on file
Dawn M. Wilson No disclosure on file
Derya U. Ugurlu, MD No disclosure on file
Puja Patel, MD No disclosure on file
Sai Krishna J. Munjampalli, MD No disclosure on file
Ketevan Berekashvili, MD Dr. Berekashvili has nothing to disclose.
Brian E. Tark, MD No disclosure on file
Andrea Lazzarotto Dr. Lazzarotto has nothing to disclose.
Angela Marsili No disclosure on file
Udai Pandey No disclosure on file
Giorgia Puorro Giorgia Puorro has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Elma Research. Giorgia Puorro has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Alexion.
Sabahattin Saip Sabahattin Saip has nothing to disclose.