Sarepta reports outcomes from trial of Duchenne muscular dystrophy therapy

Sarepta Therapeutics has announced new outcomes from its open-label Phase Ib Study 9001-103, also referred to as the ENDEAVOR trial, of adeno-associated virus (AAV)-based gene transfer therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat Duchenne …

'Making the most of every day': family runs for hope in Duchenne awareness

Sarepta seeks to expand Duchenne gene therapy Elevidys use to toddlers

Duchenne muscular dystrophy gene therapy demonstrates improved motor functions

Key takeaways: In part 2 of the EMBARK study, 8- to 9-year-old boys with DMD treated with delandistrogene moxeparvovec-rokl showed statistically significant improvements in NSAA scores and timed motor tasks compared to external controls. Functional …

'Making the most of every day' Armidale family runs for hope in Duchenne awareness

Early Data Show Potential of Tevard’s tRNA Therapy in Duchenne Muscular Dystrophy

In an oral presentation on day three of the 2025 American Society of Gene & Cell Therapy Annual Meeting (ASGCT 2025)—held May 13-17 in New Orleans—the privately held biotechnology company Tevard Biosciences presented preclinical data indicating that …

Japanese MHLW approves Chugai’s Elevidys as a gene therapy product to treat Duchenne muscular dystrophy

Chugai Pharmaceutical Co., Ltd. announced that it obtained regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) for Elevidys Intravenous Infusion [generic name: delandistrogene moxeparvovec] (hereinafter, "Elevidys"),  as a …

Duchenne Muscular Dystrophy Gene Therapy

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article, …

Navigating Duchenne Muscular Dystrophy: An Early-Childhood Case Example

Summary for Physicians Early Intervention in DMD This case highlights the importance of early diagnosis through newborn screening, enabling presymptomatic intervention in DMD. A 6-month-old infant diagnosed via screening in Ohio with an exon 51 skipping– …

Weekly review: Multiple FDA action items, USPSTF recommendation, and more

Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from the week (Monday, May 12, to Friday, May 16, 2025), and click on each link to read and watch anything you may have missed. On May 13, 2025, the FDA …