The U.S. Food and Drug Administration (FDA) has given Mesoblast the go-ahead to launch a clinical trial testing its…
Articles by Marisa Horak, MS
Boys with Duchenne muscular dystrophy (DMD) may be able to regain lost height and overcome growth stunting by switching…
Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral…
Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full approval for Amondys…
PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1…
Becker muscular dystrophy (BMD) patients who received the experimental treatment sevasemten in clinical trials had stable motor function…
Four people with oculopharyngeal muscular dystrophy (OPMD), a disease type marked by swallowing difficulties, experienced long-lasting improvements in their…
Dosing has begun in a Phase 2 clinical trial testing the oral therapy SAT-3247 in boys with Duchenne muscular…
The U.S. Food and Drug Administration (FDA) has cleared Precision Biosciences to launch a clinical trial testing its gene-editing…
Three years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial, boys with Duchenne muscular…