Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients ...

PBGENE-DMD is the first in vivo gene editing program that has the potential to transform the treatment paradigm and deliver durable functional improvement for most patients, as up to 60% of those ...

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular ...

The approval is intended for individuals with deletions in exon 8 and/or exon 9 of the DMD gene, and are negative for anti-adeno-associated virus rhesus isolate serotype 74 (AAVrh74) antibodies.

ELEVIDYS (delandistrogene moxeparvovec) is a gene therapy designed to address the genetic root of DMD by delivering a micro-dystrophin transgene to promote targeted protein production in muscle ...

PBGENE-DMD is a first-in-class in vivo gene editing approach for the majority of Duchenne Muscular Dystrophy patients impacted by dystrophin mutations in the most common ‘hot spot’ region ...

Gene therapy could be the newest breakthrough for Duchenne muscular dystrophy. While it may not be a cure, it can give independence to boys diagnosed with DMD.