News

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

The European Medicines Agency (EMA) has validated a marketing authorization application for Puldysa (idebenone) for treating respiratory dysfunction in Duchenne muscular dystrophy (DMD) patients who do not take glucocorticoids. This means that the submission, which was made as a conditional marketing authorization (potentially granted to therapies whose immediate benefit…

It’s been 15 years since Sailormen, a Popeyes Louisiana Kitchen franchise, first partnered with the Muscular Dystrophy Association (MDA) to enhance the lives of patients. It’s keeping at it, netting $681,015 in its latest efforts. The money was raised at the 15th Annual Sailormen MDA Golf Classic and the…

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

Biophytis SA is collaborating with AFM-Telethon to advance an oral pediatric formulation of Sarconeos (BIO101) for treating Duchenne muscular dystrophy (DMD). The pact calls for the French muscular dystrophy organization AFM-Telethon to give €400,000 (about $452,000) to France-based Biophytis  for further preclinical study. The funds also will go…

To help make cutting-edge genetic research more accessible, and raise awareness of rare diseases such as Duchenne muscular dystrophy (DMD), YourDNA and Cure Rare Disease (CRD) are joining forces. The initiative hopes to increase awareness about the prevalence of rare disorders in the U.S. — and about treatment…

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…

Initially approved in the United States for Duchenne muscular dystrophy (DMD) patients age 5 and older, the corticosteroid Emflaza is now available for those as young as 2. The U.S. Food and Drug Administration (FDA) recently approved PTC Therapeutics’ supplemental New Drug Application (sNDA) for the…

The prevalence rate of Duchenne muscular dystrophy (DMD) in the United States has increased, largely due to better treatments that are helping patients live longer, while the number of new cases holds steady, a study reports. Touted as the first of its kind involving DMD, the Deerfield…

Lithium chloride — once used as table salt — improved muscle size and strength in mouse models of limb-girdle muscular dystrophy D1 (LGMD1) by blocking a protein that is overactive in some people with this disease. Inhibiting this protein may be a way of correcting muscle weakness in these…