Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Parent Project Muscular Dystrophy (PPMD), in partnership with the Foundation to Eradicate Duchenne (FED), today announced Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy ...

Interim observations from the Phase 2 TRAILHEAD study show continued improvement in handgrip strength, overall stability of ...

Sen. Ron Johnson is turning up the heat on the Food and Drug Administration after parents of boys with Duchenne muscular dystrophy told him the agency's recent decisions cut off access to a drug they ...

A little boy was diagnosed with a life-limiting, muscular condition after his parents realised he was unable to jump. Kairo ...

Four patients with LGMD-R5 (gamma-sarcoglycanopathy, formerly LGMD-2C) have received the ATA-200 gene therapy as part of the ongoing Phase 1b/2 trial evaluating the safety, pharmacodynamics, and ...

Through the support of the Jett Foundation, the Criado family is working to raise funds for an accessible vehicle that could ...

Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA)New Phase 1b ...

A test of strength that raises money for a good cause. Sunday was New Bern Fire and Rescue’s annual firetruck pull.Teams of ...

Benitec Executive Chairman and Chief Executive Officer, Jerel A. Banks, M.D., Ph.D., will share an updated presentation detailing the significant, durable, improvements in dysphagic symptom burden and ...

Families affected by the condition and a dedicated support crew walked 92km across Perth over four days to raise funds and ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and ...