Mary Chapman, features writer —

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…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

With the goal of connecting with every U.S. family facing a Duchenne muscular dystrophy (DMD) diagnosis, Parent Project Muscular Dystrophy (PPMD) continues its multi-year community effort called the End Duchenne Tour. At each stop, the non-profit brings updates on research, advocacy and patient care to families in…

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…

Popeyes Louisiana Kitchen’s franchisee Sailormen has launched the 15th Annual “Appetite for a Cure” campaign to enhance the lives of those living with muscular dystrophy (MD) and related disorders.  Money raised will support efforts by the Muscular Dystrophy Association (MDA) to combat MD and other diseases of the…

Parent Project Muscular Dystrophy (PPMD) has published a report outlining key results of its decade-old study of some 4,000 registered Duchenne and Becker muscular dystrophy patients. Representing more than 100 countries, the nonprofit organization’s Ten Year Registry Report is a compilation of data provided by…

To improve patient engagement and care management tools for the Duchenne Registry, Parent Project Muscular Dystrophy (PPMD) has teamed up with THREAD, a company specializing in remote and virtual patient research. Enhancements to the registry — available on both IOS and Android devices — will be mainly…

Kanneboyina Nagaraju, PhD, and his research team at Binghamton University, State University of New York will get a $329,000 Parent Project Muscular Dystrophy (PPMD) grant to study responses to differing treatments for Duchenne muscular dystrophy. The researchers will focus on the role of the immune system…

Capricor Therapeutics put a voluntary hold on a Phase 2 clinical trial of its Duchenne muscular dystrophy (DMD) investigative cell therapy CAP-1002 after a patient experienced a severe allergic reaction during blind testing. According to a recent U.S. Securities and Exchange Commission report, Capricor halted its HOPE-2 (NCT03406780) study,…