Paul Streck, MD, Head of R&D, commented, “As of January 23, 2025, all treatment related ... for the treatment of patients with DMD amenable to an exon-51 skipping approach.

PepGen Inc. , a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological ...

CONNECT1 has enrolled two cohorts of boys and young men living with DMD amenable to exon 51 skipping and its ... “As of January 23, 2025, all treatment related adverse events in CONNECT1 have ...

Nisa Investment Advisors LLC grew its position in shares of Sarepta Therapeutics, Inc. (NASDAQ:SRPT – Free Report) by 23.1% ...

Key Insights The projected fair value for PepGen is US$14.23 based on 2 ... for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach.

The remaining 46 patients were divided into three groups: Group 1 (multiplex ligation-dependent probe amplification–negative Duchenne muscular dystrophy ... was performed using the SureSelect Human ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...