Session Details

Session Details

10:15 AM - 12:00 PM - Room 307-308

Chunping Qiao - PhD - Novartis Gene Therapies

Co-Chair

10:15 AM - 12:00 PM - Room 307-308

Carsten Bonnemann - MD - NINDS/NIH

Co-Chair

10:15 AM - 10:30 AM - Room 307-308

David Mack - PhD - University of Washington

394: Ten-Year Durability of AAV8-MTM1 Gene Transfer in a Canine Model of X-Linked Myotubular Myopathy

10:15 AM - 12:00 PM - Room 307-308

Markus Ruegg -

Co-Chair

10:30 AM - 10:45 AM - Room 307-308

Jahannaz Dastgir - REGENXBIO Inc.

395: RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data

10:45 AM - 11:00 AM - Room 307-308

Emily Walsh - Tremont Therapeutics Consulting

396: Sustained Clinical Effects of Single-Dose Intraarticular PCRX201 Injection in Moderate-to-Severe Osteoarthritis of the Knee

11:00 AM - 11:15 AM - Room 307-308

Nicolas Wein - PhD - Abigail Wexner Research Institute at Nationwide Children's Hospital

397: Correction of Myotonia and Reduction of Toxic DMPK Foci After Treatment of the HSALR Mouse Model of Myotonic Dystrophy Type 1 Using an Adeno-Associated Virus Delivering U7snRNA Interfering with CTG Repeat Expansion

11:15 AM - 11:30 AM - Room 307-308

Gretchen Thomsen - Insmed Gene Therapy

398: CSF Delivery of INS1201 AAV9-Micro-Dystrophin Demonstrates Efficacy in a Mouse Model of DMD

11:30 AM - 11:45 AM - Room 307-308

Randolph Qian - PhD - REGENXBIO Inc.

399: Evaluation of Novel Muscle-Specific Promoters in Mice and Non-Human Primates

11:45 AM - 12:00 PM - Room 307-308

Fiona Knapman - NeuRA

400: An Optogenetics-Based Gene Therapy for Obstructive Sleep Apnea