New study calls for more inclusive patient data in rare disease research
A new review finds rare disease studies often record only age and sex, missing key demographic factors that shape patients’ care.
News
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Study finds gene therapy heart risks are rare and manageable
Heart inflammation linked to gene therapy for Duchenne muscular dystrophy (DMD) is uncommon and usually mild and reversible.
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New review highlights progress and cautions in AAV gene therapy for DMD
Gene therapy for Duchenne muscular dystrophy (DMD) is advancing quickly, but important safety risks still need to be carefully managed.
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DMD worsens in young adulthood, but many report good quality of life
Arm strength and breathing get worse quickly when patients with DMD become adults, but many still report a good quality of life.
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2026 Muscular Dystrophy Association conference highlights recent advancements
Researchers highlighted recent advances in DMD research at the Muscular Dystrophy Association’s Clinical & Scientific Conference.
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New data on Deramiocel shows significant cardiac benefits in DMD
Dermamiocel appears to effectively prevent cardiac scarring (fibrosis) in patients with Duchenne muscular dystrophy (DMD).
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New steroid alternative helps boys with Duchenne maintain growth
The steroid alternative vamorolone appeared safe and effective in boys with Duchenne muscular dystrophy (DMD) aged 7 to 17.
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Gene therapy GNT0004 shows lasting benefit for boys with Duchenne
A one-time gene therapy was linked to stable or improved movement in boys with Duchenne muscular dystrophy.
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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.
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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.
