Session Details

Session Details

01:30 PM - 03:15 PM - Ballroom 1

Diana Bharucha-Goebel - MD - NIH/NINDS and Children's National Hospital

Co-Chair

01:30 PM - 03:15 PM - Ballroom 1

Christian Buchholz - PhD - Paul-Ehrlich-Institut

Co-Chair

01:30 PM - 01:45 PM - Ballroom 1

John Mitchell - Montreal Children’s Hospital

143: Long-Term Efficacy and Safety in Adults with Glycogen Storage Disease Type IA (GSD IA) from a Phase 1/2 Clinical Trial and Long-Term Follow-Up Study of DTX401, an AAV8-Mediated, Liver-Directed Gene Therapy

02:00 PM - 02:15 PM - Ballroom 1

Shin-Shay Tian - Shanghai Vitalgen Biopharma Co.

145: VGN-R08b Gene Therapy for Neuronopathic Gaucher Disease

02:15 PM - 02:30 PM - Ballroom 1

Alison Heald - MD - ICM Co., LTD

146: Interim Review of Safety, Tolerability, and Efficacy From a First-In-Human Phase 1/2a Clinical Study of ICM-203, an Intra-Articular, AAV Gene Therapy for Osteoarthritis

02:30 PM - 02:45 PM - Ballroom 1

Jerry Vockley - MD, PhD - UPMC Children's Hospital of Pittsburgh

147: Demonstration of Nuclease-Free Genome Editing (GeneRide™) in Pediatric Patients with Methylmalonic Acidemia

02:45 PM - 03:00 PM - Ballroom 1

Terence Flotte - MD - UMass Chan Medical School

148: Phase 1/2 Trial of Combined Intrathalamic/Intracisternal/Intrathecal Gene Therapy for Tay-Sachs and Sandhoff Diseases

03:00 PM - 03:15 PM - Ballroom 1

Shiyi Yin - Postgraduate - Beijing Tongren Hospital, Capital Medical University

149: Subretinal Gene Therapy in Patients with Bietti Crystalline Dystrophy: Preliminary Results of a Phase 1/2 Clinical Trial