Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients with baseline cardiomyopathy (p=0.017)Global Statistical ...

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 ...

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

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 ...

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 ...

Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular ...

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 ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

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