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

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 ...

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 ...

Analyst Joseph Pantginis of H.C. Wainwright reiterated a Buy rating on Astria Therapeutics (ATXS – Research Report), retaining the price ...

Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene and is characterized by skeletal and cardiac muscle disease. The progression of skeletal muscle disease from fibrosis to ...

Muscular dystrophy cannot be cured and there are no treatments to stop the progression of the disease. The goal is to manage symptoms and help a person to live well with the condition. Duchenne ...

The agency is open to our plan for further investigation of biomarkers, including caudate atrophy as an endpoint to evaluate ...

Good morning, and welcome to the Wave Life Sciences Third Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen only mode. As a reminder, this call is being ...

In-shoe sensors, AI and biomarkers will monitor muscular dystrophy and atrophy in children, adolescents and adults — with ...