News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to PF-06939926, Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD). This designation is given to treatments that show considerable potential in addressing serious conditions for which available treatments fall short. It is…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, Solid Biosciences’ investigational gene therapy for people with Duchenne muscular dystrophy (DMD), the company announced. Updated safety and effectiveness data for all patients dosed to date,…

A single dose of SRP-9001, Sarepta Therapeutics’ investigational gene therapy for  Duchenne muscular dystrophy (DMD), continues to be safe and to improve motor function in four boys treated two years ago, according to data from an ongoing Phase 1/2 trial. Updated results from Study-101 (…

Italfarmaco has now completed recruitment — delayed in the spring due to the COVID-19 pandemic — of boys with Duchenne muscular dystrophy (DMD) for its ongoing Phase 3 trial of givinostat, according to a top company officer. In another milestone, Givinostat has “just received” rare pediatric disease…

It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they found out Levi has Jansen’s metaphyseal chondrodysplasia — an extremely rare form of dwarfism.   “The hardest…

The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting their growth and with fewer side effects compared to standard corticosteroids, according to 18-month data from a Phase 2 trial and its extension study. Findings were reported in the study, “…

In a Facebook Live event hosted by the Muscular Dystrophy Association (MDA), experts discussed the effect that the COVID-19 pandemic has had on research efforts in muscular dystrophy, as well as the ongoing development of a potential COVID-19 vaccine. Clinical trials are a critical…

Real-world and natural history data could be used to supplement, or perhaps replace, the use of a placebo in future clinical trials in people with Duchenne muscular dystrophy (DMD), a new study suggests. The study, “Suitability of external controls for drug evaluation in Duchenne muscular dystrophy,”…

Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) that clarified regulatory requirements to start the company’s next clinical trial of SRP-9001, an investigational gene therapy for Duchenne muscular dystrophy (DMD). During the type C “written response only” meeting, the…