Dec 17, 2024 · “This landmark study presents compelling biomarker data and promising signals that suggest the potential for functional stabilization with administration of sevasemten,” Craig …

Oct 10, 2024 · After three years of treatment in the extension study, data showed sustained benefits in heart and arm function compared with the decline seen among untreated patients.. …

Apr 18, 2023 · The results showed that dystrophin protein levels increased to a mean of 10.27% of normal in patients treated with the low dose, and to 15.79% of normal in those treated with …

Jun 17, 2024 · A physical therapist knowledgeable about DMD and progressive muscle weakness and other DMD symptoms can create a therapeutic exercise program that safely stretches and …

Nov 19, 2024 · Gene therapies for MD. Muscular dystrophy refers to a group of diseases that cause progressive muscle weakness and wasting. DMD, the most common type of muscular …

Jun 21, 2024 · Assistive technologies. Advancements in technology have been a great help to people living with DMD, from enhancing accessibility with eye-tracking devices, smart …

Jul 4, 2024 · In Duchenne MD, mutations lead to a lack of functional dystrophin, a protein that normally helps protect muscles from damage.That lack of dystrophin causes muscles to …

Oct 15, 2024 · The trial, cleared by U.S. regulators last year, is evaluating BB-301’s safety and its effects on swallowing in OPMD patients.In its Phase 1b part, patients are being treated …

Nov 12, 2024 · SRP-5051 was a next-generation successor to Exondys 51 (eteplirsen), an older exon 51-skipping therapy also from Sarepta. Exondys 51 is conditionally approved in the U.S. …

Oct 22, 2024 · About a year ago, the CHMP issued a negative opinion recommending that Translarna’s conditional approval not be renewed, which would effectively pull it from the …