SIDEROS DMD Clinical Trial Seeks Participants

Researchers at the University of Kansas and Arkansas Children’s Hospital are looking for boys and men age 10 years and older with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial. The study, called SIDEROS, is designed to help researchers determine the safety and efficacy of idebenone (brand name Raxone), an experimental . . .

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Ethan Lybrand, MDA Summer Camp, and His Wheelchair Named “Bumblebee”

Ethan Lybrand loves his Bumblebee. On Oct. 27, the 7-year-old became the proud owner of his first power wheelchair. Black and yellow, the chair’s color scheme is inspired by, and named after, his favorite Transformers character, Bumblebee. For Ethan, who has Duchenne muscular dystrophy, getting a wheelchair could have been a tough transition. Instead, Ethan . . .

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Participants Sought for Clinical Trial to Test Vamorolone in DMD

Researchers are seeking volunteers to participate in a clinical trial sponsored by ReveraGen BioPharma to test the safety and effectiveness of the investigational drug vamorolone in boys with Duchenne muscular dystrophy (DMD). In the two-part, open-label study called “A Phase 2a and 2a Extension study to Assess Vamorolone in Boys with Duchenne Muscular Dystrophy,” all . . .

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Accessing Deflazacort: What You Need to Know

The U.S. Food and Drug Administration (FDA) in August accepted Marathon Pharmaceuticals’ New Drug Application for deflazacort for the treatment of Duchenne muscular dystrophy (DMD) and granted Priority Review. A decision on deflazacort, which previously has received Fast Track status, Orphan Drug designation and Rare Pediatric Disease status from the FDA, is anticipated in February . . .

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Simply Stated: What is Muscular Dystrophy?

Muscular dystrophy is a term that refers to a number of diseases that cause progressive loss of muscle mass resulting in weakness and, sometimes, loss of mobility. There are many different kinds of muscular dystrophy, each affecting different groups of muscles. In some types of muscular dystrophy, symptoms begin in childhood. In other forms, symptom . . .

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First Appeal for FDA to Review Translarna Denied

PTC Therapeutics shared the latest news today on efforts to make its experimental drug Translarna available in the U.S. for the treatment of some forms of Duchenne muscular dystrophy (DMD). The company reported that the U.S. Food and Drug Administration (FDA) has denied its first appeal that a New Drug Application for Translarna be accepted . . .

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Top 5 FAQs: Eteplirsen (Exondys 51) for DMD Treatment

On Sept. 19, the U.S. Food and Drug Administration granted accelerated approval to eteplirsen, which will now be marketed under the brand name Exondys 51 for the treatment of some forms of Duchenne muscular dystrophy (DMD). We’ve received a lot of questions about the newly approved drug, and wanted to share a few answers to . . .

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