PARALLEL SESSION 4B: Neuromuscular
Tracks
SKALKOTAS HALL
| Friday, September 20, 2019 |
| 10:30 AM - 12:15 PM |
| SKALKOTAS HALL |
Speaker
Prof. Francesco Muntoni
Great Ormond Street Institute of Child Health, UCL, NHS
Neuromuscular: outcomes in gene therapy and Ethics
10:30 AM - 10:55 AM
Dr. Kevin A. Strauss
Clinic for Special Children
OC066 | Onasemnogene Abeparvovec Gene-Replacement Therapy in presymptomatic Spinal Muscular Atrophy (SMA)
Dr. Omar Dabbous
Avexis, Inc.
OC067 | The value of AVXS-101 Gene Replacement Therapy in improving survival and motor function and decreasing ventilatory support and hospitalization contrasted to Nusinersen and natural history for Type I Spinal Muscular Atrophy
Prof. Vedrana Milic Rasic
Clinic For Neurology And Psychiatry For Children And Youth, Faculty Of Medicine, University Of Belgrade
OC068 | Joint effect of the SMN2 and SERF1A genes on early onset Spinal Muscular Atrophy patients
Ms. Georgia Wright
Clinical School, University Of Cambridge
OC069 | Clinical and radiological characterization of FIG4 related Neuronopathy
Prof. Eugenio Mercuri
Department Of Pediatric Neurology, Catholic University, Rome, Italy
OC070 | Onasemnogene Abeparvovec Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1: global pivotal Phase 3 study programme (STR1VE-US, STR1VE-EU, STR1VE-AP)
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Discussion
12:10 PM - 12:15 PMChair
Basil T. Darras
Department of Neurology, Harvard Medical School, Boston Children's Hospital
Virginia Theodorou
Neurology Department, "Aghia Sophia" Children's Hospital
