Sarepta Therapeutics, Inc. (SRPT) was founded in 1980 and is based in Cambridge, Massachusetts. The company is focused on developing RNA-based therapeutics and gene therapy against Duchenne muscular ...
Dyne Therapeutics' early trial results for DM1 and DMD treatments show dose-dependent improvements, but data is preliminary. Despite promising DYNE-101 and DYNE-251 outcomes, limited participant ...
- 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 ...
More than 30 types of muscular dystrophy have been identified. They all cause problems with movement due to muscle protein defects caused by genetic mutations. Muscular dystrophy is a group of ...
Muscular dystrophy refers to a group of more than 30 conditions, all of which cause progressive muscle weakness and loss. Some muscular dystrophies progress slowly, and people with those conditions ...
Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...
Muscular dystrophy is a group of disorders that involve a progressive loss of muscle mass and consequent loss of strength. In general, the condition is caused by genetic variations that interfere with ...
Becker muscular dystrophy (BMD) is a type of muscular dystrophy, a genetic condition that weakens and damages muscles. It can worsen with age. However, its symptoms are less severe than those of ...
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