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.

Trade/B2B, Consumer

English

Online/Digital

Outlet metrics

Domain Authority

Ranking

Global

#970048

United States

#335972

Health/Health Conditions and Concerns

#958

Traffic sources

Monthly visitors

Articles

PBGENE-DMD therapy gets FDA's rare pediatric disease status

2 days ago | musculardystrophynews.com | Marisa Wexler

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Precision Biosciences. The designation provides companies with extra incentives to develop experimental therapies to treat rare diseases that mainly affect children.
FDA advisory committee meeting for deramiocel in Duchenne MD set for July

4 days ago | musculardystrophynews.com | Marisa Wexler

The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting next month to review the data on deramiocel, an experimental cell therapy up for approval to treat heart disease in people with Duchenne muscular dystrophy (DMD). The FDA has informed Capricor Therapeutics the meeting should happen July 30, though Capricor said that date hasn’t been confirmed.
Elevidys suspended in nonambulatory DMD after second death reported

1 week ago | musculardystrophynews.com | Marisa Wexler

A second person with Duchenne muscular dystrophy (DMD) has died of acute liver failure after being treated with the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl). A first death was announced in March and both occurred in people with DMD who were no longer able to walk. Following the deaths, the gene therapy’s developer Sarepta is overhauling its safety plans for Elevidys.
Viral vector in LGMD2E gene therapy gets new FDA designation

2 weeks ago | musculardystrophynews.com | Marisa Wexler

The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an investigational gene therapy for limb-girdle muscular dystrophy type 2E (LGMD2E).
FDA grants orphan drug status to DMD cell therapy for heart issues

3 weeks ago | musculardystrophynews.com | Andrea Lobo

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart’s experimental stem cell therapy for heart problems in Duchenne muscular dystrophy (DMD), the company announced in an email sent to Muscular Dystrophy News Today.
Pepgen discontinues development of Duchenne MD exon-skipping therapy

3 weeks 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

1 month 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

1 month 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.