A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

We might expect that for an X-linked lethal recessive disorder (e.g. Duchenne Muscular Dystrophy ... light on how the underlying genetic defect leads to diverse congenital anomalies and ...

Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta ...

Long QT and congenital heart defects. This clinic also focuses on cardiovascular complaints of patients with genetic syndromes such as Marfan syndrome, Loeys-Dietz syndrome, Duchenne Muscular ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting in morning trading.

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 weeks compared with sham in patients aged 2 to 18 years with spinal muscular ...