PepGen Inc. achieved positive data from the phase 2 CONNECT1 study, using PGN-EDO51 for the treatment of exon 51 skipping DMD patients; higher 10 mg/kg dosing of drug data is expected in early 2025.
Breakthroughs, discoveries, and DIY tips sent every weekday. Terms of Service and Privacy Policy. Hundreds of children per year are born with Duchenne muscular ...
Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of PepGen’s lead Duchenne muscular dystrophy (DMD) candidate has resulted in limited ...
Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with ...
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Dyne’s DMD trial advances patient mobility and dystrophin expression
Patients with Duchenne muscular dystrophy (DMD) have seen an “unprecedented and sustained” functional improvement in a Phase ...
Sarepta Therapeutics' next Duchenne muscular dystrophy (DMD) med seems to be doing the job of spurring dystrophin expression. But do any of the data for SRP-5051 matter given the recent approval of ...
Duchenne muscular dystrophy (DMD), the most lethal musculoskeletal disease, is caused by mutations in the X-linked gene dystrophin. Patients with DMD show delayed motor milestone development at 2-6 ...
Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new ...
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