Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

Enlarged calves and progression of the pelvifemoral ... phosphokinasemia have been shown to occur in the Duchenne type of muscular dystrophy before any detectable signs of muscle weakness, 3 ...

A clinical trial investigating oral zinc supplementation in boys with Duchenne muscular dystrophy (DMD ... offering insights into disease progression and potential interventions.

A small trial of an experimental vaccine has shown promising results for patients with the most aggressive type of breast ...

Specifically, StitchR restored the protein Dystrophin, which is absent in patients with Duchenne muscular dystrophy, and the ...

The company’s progression in the Phase 1 study of SAT-3247 ... the company is advancing to the multiple-ascending-dose arm and plans to initiate a study in Duchenne muscular dystrophy patients by the ...

Capricor Therapeutics' bid to find a partner for its Duchenne muscular dystrophy (DMD ... phase 2 data showed a strong effect on disease progression. Initial results from the 20-patient HOPE ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported ...

Investors consider Dyne Therapeutics' treatments for muscle diseases and strong cash position as a speculative Buy ...

In the phase 3 EPIDYS study, Duvyzat was shown to slow down disease progression ... Project Muscular Dystrophy (PPMD), said the approval of Duvyzat “brings great hope for the Duchenne community ...

Background: Duchenne muscular dystrophy (DMD) is an X-linked, progressive neuromuscular disorder characterized by cardiac and skeletal myopathy. The cardiac and skeletal muscle manifestations have ...