S51 - Child Neurology: Bench to Bedside: Progress in Treating Genetic Disorders
| Event Time: | Thursday May 9, 2019 3:30 pm to 5:30 pm |
| Topic(s): | Child Neurology and Developmental Neurology |
| Director(s): | |
| Description: | |
| Completion Message: | |
| CME Credits: | 2 |
| Core Competencies: |
| Start/End Time | Title | Faculty |
|---|---|---|
| 5:10 PM - 5:30 PM | Q&A Panel with Authors | Faculty |
| Speaker | Disclosure |
|---|
| Start Time | Pub. | Title | Presenter |
|---|---|---|---|
| 3:30 PM | 001 | MGTA-456, A First-in-Class Cell Therapy, Enhances Speed and Level of Human Microglia Engraftment in the Brains of Transplanted Mice |
Disclosure: Dr. Goncalves has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Magenta Therapeutics. Dr. Goncalves holds stock and/or stock options in Magenta Therapeutics which sponsored research in which Dr. Goncalves was involved as an investigator.
|
| 3:30 PM | 002 | Rett syndrome gene therapy improves survival and ameliorates behavioral phenotypes in MeCP2 null |
Disclosure: Dr. Powers has nothing to disclose.
|
| 3:30 PM | 003 | Reversal of Paralysis and Prevention of Premature Death in a Mouse Germline Model of Leigh Syndrome Caused by a Mutation in Mitochondrial ATP Synathse |
Disclosure: Dr. Guy has nothing to disclose.
|
| 3:30 PM | 004 | Preservation of Function over time as Measured by North Star Ambulatory Assessment in Ambulatory Boys with Nonsense Mutation Muscular Dystrophy Treated with Ataluren |
Disclosure: Consultant on clinical trials of Duchenne muscular dystrophy (DMD) for BioMarin, Capricor, Catabasis, Eli Lilly, Italfarmaco, Mitobridge, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals, and Sarepta Therapeutics C. M. McDonald is an Editor of Muscle & Nerve and Neuromuscular Disorders. Dr. McDonald has received research support from research support for clinical trials from BioMarin, Eli Lilly, PTC Therapeutics, Santhera Pharmaceuticals, and Sarepta Therapeutics
|
| 3:30 PM | 005 | Eteplirsen-Treatment Attenuates Respiratory Decline in Ambulatory and Non-ambulatory Patients With Duchenne Muscular Dystrophy: Comparison With Natural History Cohorts |
Disclosure: Dr. Khan has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Sarepta Therapeutics, Inc. Dr. Khan has received compensation for serving on the Board of Directors of Sarepta Therapeutics, Inc.
|
| 3:30 PM | 006 | Edasalonexent, an NF-kB Inhibitor, Slows Longer-Term Disease Progression on Multiple Functional and MRI Assessments Compared to Control Period in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy |
Disclosure: Dr. Finkel has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AveXis, Biogen, Capricor, Catabasis, Ionis, Neurogene, Novartis, ReveraGen, Roche, Sarepta. . Dr. Finkel has received personal compensation in an editorial capacity for Elsevier (Swaiman's Pediatric Neurology textbook).. Dr. Finkel has received royalty, license fees, or contractual rights payments from Children's Hospital of Philadelphia (CHOP INTEND licensing fees). Dr. Finkel has received research support from AveXis, Biogen, Catabasis, Capricor, Italfaramaco, Pfizer, ReveraGen, Roche, Summit.
|
| 3:30 PM | 007 | A Novel Mouse Model with Tubb4a (D249N/D249N) for classical Hypomyelination and atrophy of basal ganglia and cerebellum |
Disclosure: Dr. Sase has nothing to disclose.
|
| 3:30 PM | 008 | Cellular models of Hypomyelination and Atrophy of Basal Ganglia and Cerebellum using rodent and human induced pluripotent stem cells |
Disclosure: Dr. Almad has nothing to disclose.
|
| 3:30 PM | 009 | 15q11.2 Deletion Results in Altered Excitability and Connectivity in Human Induced Pluripotent Stem Cell Derived Neurons |
Disclosure: Dr. Habela has nothing to disclose.
|
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