Duchenne muscular dystrophy

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.