Margarida Azevedo, MSc,  —

Wave Life Sciences, Ltd., is planning to begin clinical testing of its next-generation nucleic acid therapy for patients with Duchenne muscular dystrophy (DMD), which the company says has shown promise in pre-clinical trials. Its initial DMD candidate skips exon 51 in the dystrophin gene. Data from early studies showed a roughly…

Santhera Pharmaceuticals announced updates on regulatory filings for Raxone (idebenone), saying it plans to submit comprehensive briefing material and a meeting request to the Food and Drug Administration (FDA) to discuss filing a New Drug Application (NDA) for the treatment of Duchenne Muscular Dystrophy (DMD) patients who are not taking concomitant…

Lowe’s, a nationwide home improvement company, announced it has raised over $7.5 million across its more than 25,000 U.S. retail stores for the Muscular Dystrophy Association’s (MDA) Shamrocks program. Lowe’s is MDA’s largest Shamrocks retailer and has been involved with the program for 15 years, donating more than $57 million…

Parent Project Muscular Dystrophy (PPMD) is reaching out to families affected by Duchenne muscular dystrophy (DMD) through its ‘Every Single One’ Tour, a multi-year campaign to bring updates on disease research, care, and advocacy efforts to communities across the U.S. The campaign, whose daylong sessions are free of charge, kicked…

Parent Project Muscular Dystrophy (PPMD), the largest U.S. nonprofit group focused on Duchenne muscular dystrophy (DMD), is holding a Pulmonary Outcomes Workshop this week, April 14–15. Physicians and industry experts will discuss pulmonary outcome measures used in current DMD clinical trials, how those measures might be improved, and their use in furthering drug development.

The Muscular Dystrophy Coordinating Committee (MDCC) has released an updated version of its Action Plan for the Muscular Dystrophies, a document that works as a comprehensive guide to address the challenges experienced by people with muscular dystrophy. The committee, authorized by Congress, is composed of representatives from U.S. federal agencies and…

Researchers at Switzerland’s Basel University Hospital investigated the biochemical and physiological properties of the muscles involved in the control of the movements of the eyelids along with the extraocular muscles, which control eye movements, noting that these muscle groups express low levels of dystrophin and high levels of utrophin, a similar protein…

Marathon Pharmaceuticals recently presented findings from a Phase 3 trial comparing the effects of deflazacort to prednisone and placebo in boys with Duchenne muscular dystrophy. The presentation, showing deflazacort was more effective than prednisone in timed functional tests and in limiting weight gain, was given at the 2016 Muscular Dystrophy Association (MDA) Clinical Conference, and will be shown again at…

Marathon Pharmaceuticals has announced its investigational medicine deflazacort will be accessible for free through an Expanded Access Program (ACCESS DMD) for patients with Duchenne muscular dystrophy (DMD). Deflazacort will now be made available to eligible patients through a growing network of medical research centers across the nation. “In…

Ultragenyx, a biopharma dedicated to the development of investigational products for rare diseases, will fund the efforts of a team of scientists from Saint Louis University (SLU) to advance a treatment for muscular dystrophy, inspired by lead researcher Dr. Fran Sverdrup’s daughter, who was diagnosed with the disease. SLU’s Center…