News

Expanding its network of institutions that provide standardized, comprehensive care for Duchenne patients, the Parent Project Muscular Dystrophy (PPMD) has named Children’s National Health System its newest Certified Duchenne Care Center (CDCC). This most recent designation brings to 20 the number of clinics to pass muster since the CDCC…

The Hippo signaling pathway — a key regulator of tissue development, and cell growth and death — does not work as it should in Duchenne muscular dystrophy and acts to prevent, rather than aid, the regeneration of muscle cells in DMD patients, a study reports. A member of this pathway, a…

Capricor Therapeutics’ vesicle-based therapy CAP-2003, derived from the company’s proprietary cardiosphere-derived cells (CDCs), may be an effective therapy for Duchenne muscular dystrophy (DMD), according to preclinical data. The company presented the results in August in two scientific posters at the 2018 Gordon Research Conference on…

A campaign hopes to raise awareness of Duchenne muscular dystrophy (DMD) and promote research toward a cure by asking supporters to paint a nail on one of their little fingers blue. The Pinkies Up campaign is gearing up for November. (Photo courtesy of CureDuchenne) Called “Pinkies…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…

Muscle biopsies can benefit the diagnosis and treatment of neuromuscular diseases in children, particularly those with muscular dystrophy or inflammatory muscle disease, a study suggests. In the study, “Does muscle biopsy change the treatment of…

To raise money for treatments and to support families touched by muscular dystrophy, the Muscular Dystrophy Association’s (MDA) New York Muscle Team will present its 22nd annual gala Dec. 3 in New York City. To be held at Cipriana Wall Street, the event will feature former New York…

Taking care of patients with Duchenne muscular dystrophy (DMD) can have significant effects on caregivers, leading to a lower health‐related quality of life, poor sleep quality, depression, pain, stress, sexual dysfunction, and lower self‐esteem, a review study reports. Caregiving can also have a considerable negative impact on work life…

Researchers at The University of Western Australia (UWA) have been awarded close to $490,000 from two organizations to explore the potential use of an amino acid called taurine as a treatment for Duchenne muscular dystrophy (DMD). Duchenne UK granted £273,648 (approximately $356,515) to support the study, while Save our Sons…

The digital health firm ZappRx is working with CureDuchenne to provide faster and more efficient delivery of treatments for Duchenne muscular dystrophy to patients starting next year. ZappRx streamlines the often-complicated processes involved in ordering specialty medications by collecting and storing required information from pharmacies, insurance companies and medical providers.