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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...

Entrada has secured the authorisation within the EU Clinical Trial Regulation to begin the Phase I/II trial of ENTR-601-45.

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

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

Pepgen will stop development of its Duchenne MD exon-skipping therapy PGN-ED051 after increases in dystrophin levels proved ...

ELEVIDYS is indicated in U.S. 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.

The approved patient population are ambulatory patients with DMD who do not have a deletion of any portion or the entirety of exon 8 and/or exon 9 in ... Elevidys is the gene therapy product for ...

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