At Binghamton University, researchers were among the first to find ways to help patients with Duchenne muscular dystrophy ...
Precision BioSciences has activated Arkansas Children’s Hospital as the first site and initiated patient enrolment for its Phase I/II FUNCTION-DMD clinical trial of PBGENE-DMD for Duchenne muscular ...
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced that the Cellular, Tissue, and Gene ...
SAT-3247 has now received FDA Fast Track, Orphan Drug and Rare Pediatric Disease designations for Duchenne muscular dystrophy (“Duchenne” or ...
Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...
In yet another fail for the Duchenne muscular dystrophy (DMD) field, Taiho Pharmaceutical Co. Ltd.’s pizuglanstat (TAS-205) did not meet the primary endpoint in a phase III trial. The phase III ...
-Arkansas Children’s Hospital activated as the first clinical trial site and now enrolling patients in the FUNCTION-DMD study of PBGENE-DMD- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical ...
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
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