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2018 Annual Meeting | S29 - Child Neurology and Developmental Neurology I

Wednesday 04/25/18
01:00 PM - 03:00 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
Child Neurology and Developmental Neurology
2.00 CME credits
Event Timeline
01:00 PM - 03:00 PM Abstract 001 - AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Continued Event Free Survival and Achievement of Developmental Milestones
Jerry R. Mendell, MD, FAAN
01:00 PM - 03:00 PM Abstract 002 - AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Patients Treated Early with the Proposed Therapeutic Dose Were Able to Sit Unassisted at a Younger Age
Lindsay N. Alfano, PT
01:00 PM - 03:00 PM Abstract 003 - AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Continued Independence from Nutritional and Ventilatory Support in Patients Dosed Early in Disease Progression
Richard Shell
01:00 PM - 03:00 PM Abstract 004 - AVXS-101 Trial Experience: CHOP-INTEND Detects Early Improvements in Infants with SMA Type 1 but is not Sensitive to Continued Advances in Motor Function
Linda P. Lowes, PT PhD
01:00 PM - 03:00 PM Abstract 005 - A Subset of Axonal and Synaptic Genes with Altered Expression/Splicing in SMA-MNs Harbours a Common Motif that Interacts with SMN/SYNCRIP Complex
01:00 PM - 03:00 PM Abstract 006 - MoveDMD®: Positive Effects of Edasalonexent, an NF-?B Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy in Phase 2 Study with an Open-Label Extension
Richard S. Finkel, MD, FAAN
01:00 PM - 03:00 PM Abstract 007 - Efficacy and Safety of OnabotulinumtoxinA for the Treatment of Pediatric Lower Limb Spasticity: Primary Results
01:00 PM - 03:00 PM Abstract 008 - Gene Therapy in Children with AADC Deficiency with AGIL-AADC Leads to De Novo Dopamine Production and Sustained Improvement in Motor Milestones over 5 Years
02:35 PM - 03:00 PM Discussion Discussion
Faculty Disclosures
No disclosure on file
Nereo Bresolin, DR No disclosure on file
Richard S. Finkel, MD, FAAN Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Capricor. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ReveraGen. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Dr. Finkel 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. The institution of Dr. Finkel has received research support from AveXis. The institution of Dr. Finkel has received research support from Biogen. The institution of Dr. Finkel has received research support from Capricor. The institution of Dr. Finkel has received research support from Catabasis. The institution of Dr. Finkel has received research support from ReveraGen. The institution of Dr. Finkel has received research support from Roche. The institution of Dr. Finkel has received research support from Scholar Rock. Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. Dr. Finkel has received personal compensation in the range of $0-$499 for serving as a Speaker in workshop with National Academy of Sciences. Dr. Finkel has a non-compensated relationship as a advisor with n-Lorem Foundation that is relevant to AAN interests or activities. Dr. Finkel has a non-compensated relationship as a Board Member with EveryLife Foundation that is relevant to AAN interests or activities.
John T. Kissel, MD, FAAN Dr. Kissel 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.
Arthur H. Burghes, MD No disclosure on file
Gihan Tennekoon, MD No disclosure on file
Stefania Corti, MD, PhD Dr. Corti has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Corti has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Corti has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta.
Kirsten L. Gruis, 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.
Rozalina Dimitrova, MD, MPH No disclosure on file
Erika L. Finanger, MD Dr. Finanger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics. Dr. Finanger has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Dr. Finanger has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Reata. Dr. Finanger has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer.
Brian K. Kaspar, PhD No disclosure on file
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.
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.
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.
Linda P. Lowes, PT PhD The institution of Ms. Lowes has received research support from Sarepta Therapeutics.
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.
Elizabeth A. Kichula, MD, PhD No disclosure on file
Giacomo Comi Giacomo Comi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Giacomo Comi has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis.
Katherine L. Berry, MD No disclosure on file
Joanne Donovan, MD, PhD No disclosure on file
Sukumar Nagendran, MD No disclosure on file
Anne Marie Conway, RN, MHA No disclosure on file
Megan Iammarino, PT Dr. Iammarino has nothing to disclose.
Richard Shell No disclosure on file
Maria Mancini No disclosure on file
Monica Nizzardo No disclosure on file
Kathleen Church No disclosure on file