Sarepta voluntarily pauses Elevidys shipments in the US
Sarepta Therapeutics has announced that it will voluntarily stop all shipments of the DMD therapeutic agent Elevidys.
News
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New study identifies simple test to predict fall risk in children with DMD
New research has found that the the Four-Square Step Test can help identify children with DMD at risk of frequent falls
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New study provides insights into beta-blocker therapy efficacy in DMD
Beta-blocker therapy appears to be beneficial in the context of Duchenne muscular dystrophy (DMD) complicated by cardiac dysfunction.
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FDA rejects Capricor Therapeutics’ application for DMD cell therapy Deramiocel
Caprior Therapeutics noted it will work with the FDA to resolve outstanding issues required for the progress of Deramiocel.
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Developmental delays should prompt testing for DMD, study suggests
DMD can sometimes manifest initially in children as developmental delays.
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Study finds new corticosteroid for DMD improves mobility with fewer side effects
Vamorolone can offer the same benefits as traditional glucocorticoid medications used to manage DMD, but with fewer side effects.
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Micro-dystrophin gene therapy improves survival but may pose long-term risks
Micro-dystrophin gene therapy greatly extended lifespan and improved heart function in severe DMD preclinical models.
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Small molecule drug improves muscle health in DMD mice
A recent study found that inhibiting ADAMTS-5, a cartilage-degrading enzyme, with GLPG1972 supports muscle function in a mouse model of DMD.
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PBGENE-DMD receives Rare Pediatric Disease Designation from FDA
PBGENE-DMD could help up to 60% of patients with Duchenne muscular dystrophy by permanently editing their dystrophin gene.
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New study calls for more awareness of mental health problems in DMD
DMD is often seen as a muscle disease, but new research shows many with DMD also face mental health and neurodevelopmental challenges.
