Duchenne muscular dystrophy

A child died in a Chinese gene-editing trial for Duchenne muscular dystrophy conducted by HuidaGene, reigniting concerns about transparency and safety in such trials. The company presented initial data at a U.S. conference but later became unresponsive, with its CEO and CTO departing, and the study was recently declared complete without clear details on patient outcomes.

A Suffolk family is seeking community assistance after their wheelchair accessible van broke down, jeopardizing an upcoming trip for Kaden Dickerson, who has Duchenne Muscular Dystrophy and relies on his wheelchair and medical equipment for travel.

The article discusses the FDA's inconsistent approaches to Duchenne muscular dystrophy drugs, potential changes in drug company leadership, and Adam's conversation with a recruiter. It references a podcast episode exploring these topics.

An FDA advisory panel rejected a Duchenne muscular dystrophy drug by a 9-to-3 vote due to insufficient effectiveness data, and the FTC sued Hims & Hers over alleged misleading health data practices and subscription billing issues.

An FDA panel voted 9-3 to reject the investigational drug deramiocel for treating cardiomyopathy in Duchenne muscular dystrophy patients due to insufficient evidence of effectiveness.

Sarepta Therapeutics appointed Michael Severino, formerly CEO of Tessera Therapeutics, as its new chief executive. The company, which has three Duchenne muscular dystrophy treatments approved, faces ongoing questions about their effectiveness and regulatory scrutiny over gene therapy safety, contributing to a decline in its share price.

Sonothera, a California startup, claims its ultrasound technology could treat Duchenne muscular dystrophy and other genetic diseases, though it lacks clinical data. Animal study results presented by the company have sparked skepticism from experts like Eric Olson and Jeffrey Chamberlain, who question the feasibility of the claims.

An 18-year-old Illinois teen with Duchenne muscular dystrophy, Brent Meizelis, fulfilled his dream of interacting with dolphins in Key Largo through a collaboration between Make-A-Wish Illinois and Make-A-Wish Southern Florida. He participated in dolphin care activities, swam with the animals, and expressed gratitude for the experience, which his mother described as creating lifelong memories.

Regenxbio announced it will submit its Duchenne muscular dystrophy gene therapy for accelerated approval, despite the FDA previously requesting an additional trial. The FDA appears to be reversing recent rejections of gene therapies, including a Hunter syndrome treatment reconsidered after key officials, Marty Makary and Vinay Prasad, left the agency.

French pharma company Servier will acquire Edgewise Therapeutics’ muscular dystrophy business in a deal worth up to $2.65 billion. The acquisition centers on Edgewise’s sevasemten, an oral drug in pivotal trials for Becker muscular dystrophy and Phase 2 trials for Duchenne muscular dystrophy.

Regenxbio's experimental gene therapy for Duchenne muscular dystrophy has produced high levels of a miniaturized muscle protein, paving the way for FDA submission. The company aims to create a safer and more effective therapy than Sarepta Therapeutics' Elevidys. Regenxbio is seeking accelerated approval.

Capricor Therapeutics accuses Nippon Shinyaku of failing to follow through on marketing plans for a Duchenne muscular dystrophy treatment. A pricing glitch in their distribution agreement would make the therapy economically impractical for patients covered by Medicare, Medicaid, and private insurers. The issue was disclosed in March 2025.

Capricor Therapeutics is suing its partner Nippon Shinyaku over the launch of its Duchenne therapy, claiming they botched preparations and created an inaccessible pricing structure. The biotech company is fighting for its promising treatment, deramiocel. This lawsuit may impact the accessibility of the treatment for patients.

Entrada Therapeutics' next-generation drug for Duchenne muscular dystrophy disappointed in an early trial, raising questions about the company's competitiveness. The drug is designed to help patients produce shortened but still functional forms of dystrophin. Scientists have devised ways to redesign these molecules for better results.

A mother caring for her son with Duchenne muscular dystrophy criticizes the FDA for regulatory delays in approving experimental drugs, arguing that bureaucratic caution harms terminally ill children. She calls for FDA Commissioner Dr. Martin Makary to address the issue and appoint a successor to Dr. Vinay Prasad who prioritizes patient access over caution.