PTC Therapeutics withdraws ataluren application after FDA review
The FDA determined that current data for ataluren in Duchenne muscular dystrophy (DMD) are unlikely to meet approval standards.
News
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Genetic clues emerge for heart damage in Duchenne muscular dystrophy
Certain genes may help explain why heart disease is worse in some people with Duchenne muscular dystrophy.
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Boys with DMD experience drop in executive function during middle years
The developmental trajectory of boys with DMD show an increase in executive function impairment with age.
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Two-year study shows lasting gains from Elevidys gene therapy for DMD
A single gene therapy dose helped young boys with Duchenne muscular dystrophy keep their strength longer than expected.
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International study reveals high caregiver burden in DMD
Caregivers of individuals with DMD report substantial disruptions to work and everyday life across the U.S., Europe and Japan, study finds.
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Researchers examine how to improve safety of gene therapy for Duchenne
Gene therapy for Duchenne muscular dystrophy (DMD) shows real promise, but high doses have caused rare serious side effects.
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Study finds environmental factors lower participation in daily life in DMD
Children with DMD take part in fewer daily activities than peers, and physical environmental obstacles appear to be the key factor.
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Study reveals effects of work and income disruptions among DMD caregivers
Caregivers of individuals with DMD face substantial impacts on employment, productivity and income, new research finds.
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Case report: Gastric dilation in a teenager with DMD
Gastrointestinal manifestations remain relatively overlooked in terms of possible symptoms of DMD.
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HHS adds Duchenne to newborn screening panel
Adding DMD and metachromatic leukodystrophy to newborn screening can help identify affected babies before symptoms appear.
