Muscular Dystrophy News

Muscular Dystrophy News is a daily online news platform dedicated to providing patients with muscular dystrophy and their families the most recent updates on scientific research and advancements related to the condition. Although there is significant research focused on treating muscular dystrophy, health media often overlooks this crucial information. MD News aims to bridge this gap in health coverage by utilizing our skilled team of researchers and reporters to gather the latest news on muscular dystrophy and present it in a way that is easy for patients to comprehend.

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Pepgen discontinues development of Duchenne MD exon-skipping therapy

5 days ago | musculardystrophynews.com | Marisa Wexler

Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy for Duchenne muscular dystrophy (DMD). The company said it will wind down all DMD-related research and development activities, focusing instead on an investigational therapy for myotonic dystrophy type 1 (DM1) that’s in clinical testing.
Analyses show benefits of DMD gene therapy Elevidys

2 weeks ago | musculardystrophynews.com | Lindsey Shapiro

The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne muscular dystrophy (DMD) who were treated when they were 8 or 9 years old, according to new trial analyses.
DMD gene therapy Elevidys wins conditional approval in Japan

3 weeks ago | musculardystrophynews.com | Marisa Wexler

The Japanese Ministry of Health, Labour, and Welfare (MHLW) has conditionally approved the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat some cases of Duchenne muscular dystrophy (DMD). The approval covers its use in children, ages 3-7, who don’t have deletions in exon 8 and/or exon 9 in the DMD gene and are negative for antibodies against the viral vector AAVrh74. Conditional and time-limited, the approval allows Elevidys to be sold in Japan for up to seven years.
FDA set to decide in August on deramiocel for DMD cardiomyopathy

1 month ago | musculardystrophynews.com | Marisa Wexler

The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell therapy designed to treat heart disease in Duchenne muscular dystrophy (DMD), by the end of August. The FDA agreed this year to review an application to approve deramiocel for DMD cardiomyopathy, or disease of the heart muscle. The agency granted the application priority review, shortening the review time to six months down from the usual 10 months, with a decision expected by Aug. 31.
Myotonic dystrophy patients face high healthcare costs: US study

1 month ago | musculardystrophynews.com | Patricia Inacio

People with myotonic dystrophy (DM) use more healthcare resources and face higher associated costs than patients without the disease, according to a U.S. study that analyzed a large database of insurance claims. After diagnosis, DM patients are more likely to be hospitalized and visit an emergency department, researchers found. Factors contributing to higher care costs include the need for gait aids or respiratory support devices.
CureDuchenne set to host 2025 Futures conference in San Antonio

1 month ago | musculardystrophynews.com | Andrea Lobo

CureDuchenne will host its annual Futures National Conference May 22-25 in San Antonio, as part of an effort to provide education and offer connections to members of the Duchenne and Becker muscular dystrophy communities. Under the theme “Together We Thrive,” the event will share information on research and resources for families affected by Duchenne muscular dystrophy. Registration is open and participants can attend online or in person.
Gene therapy Elevidys has given 'hope' to DMD community, Sarepta exec says

1 month ago | musculardystrophynews.com | Marisa Wexler

It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics, was approved by the U.S. Food and Drug Administration (FDA), marking the first time a gene therapy for DMD was authorized in the U.S.Since its launch, the response to Elevidys has been “overwhelmingly positive,” James Richardson, MD, Sarepta’s chief medical officer, told Muscular Dystrophy News Today in a written Q&A.
FDA OKs trial of SRP-9005 gene therapy for LGMD type 2C

1 month ago | musculardystrophynews.com | Marisa Wexler |Patrick Moeschen |Lindsey Shapiro

The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies already in clinical testing for other types of LGMD.
A Singaporean theater production reveals caregiving complexity

2 months ago | musculardystrophynews.com | Marisa Wexler |Betty Vertin

I recently had the opportunity to attend a Singaporean theater production titled “Supervision,” thanks to the SingHealth Patient Advocacy Network (SPAN). SPAN is a collective of patients and caregivers who provide feedback to improve healthcare here in Singapore. I’ve been part of it since 2022. The invitation to the play was a thank-you for volunteering with several healthcare projects, including reviewing fact sheets on chronic illnesses for the Health Ministry’s Agency for Care Effectiveness.
Independent committee favors continuing DMD gene therapy Elevidys dosing

2 months ago | musculardystrophynews.com | Andrea Lobo

An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with Duchenne muscular dystrophy (DMD) in ongoing clinical trials, according to developer Sarepta Therapeutics. The determination comes after recruitment and dosing in certain clinical trials were paused after a young man who received the gene therapy died due to acute liver failure.