Last week, an FDA advisory committee voted in favor of approving Sarepta Therapeutics, Inc.'s SRP-9001 for treating DMD. Sarepta Therapeutics stock has good near-term momentum as the BLA is scheduled ...
The milestone was earned when Arrowhead achieved the second development milestone event in a Phase 1/2 clinical study of ...
A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in ...
-- Muscular Dystrophy Association Calls FDA Approval of Novartis' Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community Mary Fiance, National Vice ...
- BridgeBio will also share 15-month Phase 2 data and review the Phase 3 clinical trial design of BBP-418, a potential therapeutic for patients with LGMD2I, with initiation of its Phase 3 study ...
The U.S. Food and Drug Administration has approved Novartis' gene therapy for patients with a rare muscle disorder, the drugmaker said on Monday.
I’ve been reflecting on the disappointing outcomes and the reaction to Sarepta Therapeutics’ recently announced clinical ...
A person may inherit the genetic changes responsible for muscular dystrophy. These genetic changes can also occur due to spontaneous genetic mutations. In either case, the disease is not preventable.
While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...
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