Noah Stansfield

The chief medical advisor at the Muscular Dystrophy Association shared key highlights of what attendees can expect at the 2025 MDA Clinical and Scientific Conference. [WATCH TIME: 4 minutes]

Prior to the 2025 MDA Conference, the vice president of Public Policy & Advocacy at the Muscular Dystrophy Association talked about the increasing interest in gene therapy in the field of neuromuscular diseases.

According to recent studies and the latest insights provided by experts, early developments in gene and cell therapies show promise for patients living with Parkinson disease, but challenges remain.

Following a successful first patient treatment experience with TSHA-102, an adeno-associated virus vector-based gene therapy for Rett syndrome, Taysha Gene Therapies was recommended to continue with investigating the therapy in a second patient.

Three of 4 patients withdrew from their previous enzyme replacement therapy following treatment with AT845 gene therapy.

Descartes-08, an investigational autologous RNA-engineered chimeric antigen receptor T-cell therapy (rCAR-T) has been dosed with the first participant in phase 2b randomized controlled trial.

Recruitment has begun for REGENXBIO's AFFINITY BEYOND clinical trial assessing the prevalence of AAV8 antibodies in male patients with Duchenne muscular dystrophy.

In addition to meeting primary end point of change in North Star Ambulatory Assessment, treatment with SRP-9001 resulted in statistically significant improvements in timed function tests.