Mutations of the gene encoding dystrophins have long been known to cause the debilitating muscle-wasting disease DMD, which affects one in every 5,000 boys born. People with the condition…
- Publication in Human Gene Therapy provides rationale for the use of microdystrophin expression levels as a surrogate endpoint reasonably likely to predict clinical benefit for AAV gene…
By IANS
MUMBAI: Vishal Jethwa, who plays a terminally ill boy in the upcoming Kajol-starrer 'Salaam Venky', watched videos of people living with DMD (Duchenne…
BOSTON, Oct. 13, 2022 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of…
- Poster Highlights In Vivo Data for DYNE-251, Now Being Evaluated in Phase 1/2 DELIVER Clinical Trial in Patients Amenable to Exon 51 Skipping - - First Presentation of In Vitro Data for…
CAMBRIDGE, Mass., Sept. 29, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has…
Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered…