Duchenne muscular dystrophy (DMD) has long been one of the most devastating genetic disorders to affect children. Marked by progressive muscle degeneration, early loss of mobility, and eventual ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

ELEVIDYS is approved for individuals ages 3- to less than 8-years-old, who do not have any deletions in exon ... in advancing the treatment of Duchenne muscular dystrophy through innovative ...

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

The approved patient population are ambulatory patients with DMD who do not have a deletion of any portion or the entirety ... Elevidys is the gene therapy product for Duchenne muscular dystrophy (DMD ...

for the treatment of Duchenne muscular dystrophy (DMD) under the conditional and time-limited approval pathway in Japan. ELEVIDYS is approved for individuals ages 3- to less than 8-years-old, who do ...

In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD ... infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene.

PepGen ends Duchenne drug program after weak Phase 2 results, pivots to DM1 treatment showing early signs of splicing ...

Two index cases with both Duchenne muscular dystrophy and autism spectrum disorder ... In addition, five of the eight boys showed deletion in the dystrophin gene on polymerase chain reaction.