(Z)-Endoxifen receives Rare Pediatric Disease designation from FDA
(Z)-Endoxifen, a drug being developed by Atossa Therapeutics for DMD, recently received Rare Pediatric Disease Designation from the FDA.
News
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New data indicates DYNE-251 boosts dystrophin and preserves function
A new treatment candidate for DMD increased dystrophin levels and improved movement while keeping lung function steady.
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New findings indicate that DMD begins to effect muscle in utero
A better grasp of the molecular mechanisms underpinning DMD may accelerate therapeutic development.
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Deramiocel trial yields positive results in patients with DMD
Findings from the HOPE-3 trial indicate that deramiocel may improve cardiac and skeletal muscle health in patients with DMD.
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Obestatin alleviates symptoms and improves muscle health in DMD models
In mouse and human cell models of DMD, treatment with obestatin repaired muscle damage and improved cellular function.
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Glucocorticoids found to improve thinking ability in a Duchenne mouse model
A steroid improved memory and brain cell connections in a mouse model of Duchenne muscular dystrophy (DMD).
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Hand X-ray measurements could help assess bone health in DMD
Bone health index and bone age may be practical, noninvasive tools that could complement DXA in clinical care for DMD, researchers say.
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Sarepta advances study aimed at reducing liver injury risk in gene therapy
The FDA authorized Sarepta to test whether using sirolimus before gene therapy can lower the risk of acute liver injury in nonambulatory DMD.
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Targeted gene therapy boosts breathing strength in Duchenne model
Targeting breathing muscles improved dystrophin levels and reduced damage with fewer liver risks in a mouse model of DMD.
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Video-based rehabilitation helps children with DMD breathe better
Doing pulmonary rehabilitation through telemedicine for three months can greatly improve breathing in children with DMD.
