Mary Chapman, features writer —

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…

A FinancialForce platform has allowed the Muscular Dystrophy Association (MDA) to enhance fundraising efforts while streamlining accounting activities. Since completing the platform launch and implementing an administrative realignment, the MDA has 40% more time to focus on fundraising strategy, according to a FinancialForce press release. Divisions such…

Joey Chorzewski has Duchenne muscular dystrophy, a neuromuscular disease that makes him different from most of his peers in Marshfield, Massachusetts. But in other ways — like wanting to wear the same kind of clothing his friends wear — he’s a typical teenager. Thanks in part to the nonprofit …

While federal funding for fiscal year 2020 has not been finalized, 79 members of Congress have vowed to back Duchenne muscular dystrophy (DMD) research, patient support efforts and drug development. On its website, Parent Project Muscular Dystrophy (PPMD) heralded results of its annual bipartisan campaign, expressing optimism…

Patients with Duchenne muscular dystrophy (DMD) and their caregivers now have a new tool to use to find an array of goods, programs, and services that can assist them with a variety of needs. The resource finder is part of the Duchenne Family Assistance Program (DFAP), an effort the…

Building on their international trade association’s relationship with the Muscular Dystrophy Association (MDA), two Virginia firefighters will set out on a 130-mile hike this month to raise awareness of the partnership, and to promote newborn screening for neuromuscular conditions. The firefighters — medic Joe Jarman and lieutenant Travis…

To raise awareness and money to back efforts that help those living with muscular dystrophy (MD), the Muscular Dystrophy Association (MDA) has launched its venerable #MDAShamrocks campaign. More than 20,000 businesses throughout the nation are expected to participate in this, the 37th annual St. Patrick’s Day-themed program to…

With the goal of identifying patients with Duchenne muscular dystrophy (DMD) in the Middle East and North Africa, genetic diagnostics company Centogene has announced a year-long strategic collaboration with Sarepta Therapeutics. The agreement calls for patients who are multiplex litigation-dependent probe amplification (MLPA) negative to undergo…

Citing a persistent need for new treatments and patient support, the Muscular Dystrophy Association (MDA) has updated its Duchenne muscular dystrophy (DMD) fact sheet. In related news, two top issues identified in a landmark MDA-funded survey of neuromuscular disease families were access to quality care along with a greater…