Study shows givinostat is effective in patients with DMD long-term
An open-label extension of the EPIDYS clinical trial found that givinostat is safe and delays disease progression in patients with DMD.
News
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KER-065 granted FDA Orphan Drug designation for DMD
KER-065, an experimental Duchenne muscular dystrophy (DMD) drug, has been granted Orphan Drug designation by the FDA.
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Case report: Patient with DMD receives successful heart transplant
A young man with DMD received a heart transplant at 18 years old and reports positive outcomes after 3.5 years.
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Patients face new challenges as Duchenne gene therapies advance
Recent gene therapies for Duchenne muscular dystrophy offer hope but also bring risks, including patient deaths.
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Newborn screening for Duchenne muscular dystrophy offers earlier answers
Earlier detection of DMD through newborn screening could give families more time to access treatment and plan care.
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New review highlights the complexity of treating cognitive aspects of DMD
One-third of individuals with DMD experience cognitive or behavioral differences, but treating DMD’s impact on the brain poses challenges.
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Study finds postural control clearly declines in patients with DMD over a one-year period
Patients with DMD significantly lose postural control over a relatively short span of time.
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Review explores delivery of DMD therapies to the central nervous system
New RNA-based treatments show promise for improving brain symptoms in DMD by reaching the central nervous system.
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Diagnosing DMD before symptoms appear may mean better motor function later
Physicians should aim to diagnose DMD as early as possible.
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Dyne Therapeutics receives Breakthrough Therapy designation for DYNE-251
DYNE-251, developed by Dyne Therapeutics and aimed at treating DMD, has received a Breakthrough Therapy Designation.
