Capricor Therapeutics’ stock has soared by more than 370% after a pivotal trial of its Duchenne muscular dystrophy (DMD) cell therapy candidate met its endpoints. The Phase III HOPE-3 trial ...
The Collaborative Trajectory Analysis Project (cTAP) announces the publication of a novel, validated prognostic score for predicting the loss of ambulation (LoA) in patients with Duchenne muscular ...
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 ...
Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...
Health Canada Priority Review targets an expedited 180-day review period for givinostat, reflecting Italfarmaco’s commitment ...
-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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