After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...
REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
Most individuals diagnosed with a rare disease won’t live to become teenagers or adults. I knew what I had to do.
This article explores the challenges and highlights actionable telehealth solutions that enhance access to care, particularly for mothers at higher risk of postpartum depression. Maternal health ...
Newborns in Minnesota can now be screened for Duchenne muscular dystrophy (DMD) and guanidinoacetate methyltransferase (GAMT) deficiency. While there is no cure, treatment can make symptoms and ...
They say the truth is stranger than fiction, and seeing it brought into sharper focus through the lens of documentary filmmaking has given us some of our most unforgettable viewing experiences ...
About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a ...
Nick Webb tells attendees at the Association of Cancer Care Centers that consumer experience and workforce happiness will be keys to success in the future for health systems. At a conference on ...
10,000-square-foot facility to house corporate offices and serve as a center for meetings and events with members of the Duchenne muscular dystrophy and other rare disease communities "ITF ...
PepGen is “temporarily pausing” a Phase 2 study of its exon 51 skipping drug for Duchenne muscular dystrophy, it said Tuesday.
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