Margarida Azevedo, MSc,  —

The U.S. non-profit, CureDuchenne, is now making the difference in Nepal, with the organization working for about a year on site in collaboration with the Muscular Dystrophy Organization Nepal (MDON). The physical and emotional burden of Duchenne Muscular Dystrophy is experienced by patients worldwide, despite their location. Being aware of…

Privately-held biopharmaceutical company, Tarix Orphan LLC, has just announced the US Food and Drug Administration has granted its lead candidate for Duchenne Muscular Dystrophy, TXA127 (angiotensin 1-7), Fast Track Designation for its potential in lessening damage and fibrotic activity in skeletal muscle. Under a previously filed Investigational New Drug (IND)…

The Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC), at the University of Iowa (UI), has won a $7.4 million grant from the National Institute of Neurological Disorders and Stroke (NINDS), a subdivision of the National Institutes of Health (NIH), renewing its funding for five years, through 2020. The center’s key…

BioMarin Pharmaceutical, Inc. recently presented findings on Pompe disease along with two posters on Duchenne muscular dystrophy and Pompe disease during the ongoing 20th International Congress of the World Muscle Society in Brighton, England, which started September 30 and ran until October 4. The oral presentation on Pompe disease included preliminary…

Parent Project Muscular Dystrophy (PPMD), the largest and most comprehensive nonprofit organization in the US focused on finding a cure for Duchenne muscular dystrophy, recently announced it will collaborate with Sarepta Therapeutics, Inc, BioMarin Pharmaceutical Inc. and PTC Therapeutics for the upcoming phase of the very successful Decode Duchenne program.

One of the country’s leading not-for-profit organizations for Duchenne Muscular Dystrophy, CureDuchenne, recently launched a certification program for physical therapists, aiming to equip these members of the health force with specialized training in dealing with patients with DMD. The CureDuchenne Cares Physical Therapist Certification Program is set to begin in 2016,…

A recent special issue of the BioMed Research International journal was published focusing on the newest clues of the mechanisms behind muscular dystrophies and novel directed therapies for the disease. There are various forms of muscular dystrophy and, as the name implies, they are characterized by wasting of the muscles throughout the…

The causes behind Duchenne muscular dystrophy (DMD) are not fully understood and, as in most muscular dystrophies, no current cure or specific treatment exists. As such, management of affected patients involves corticoids and is directed to the complications of the disease, namely physical therapy for muscle weakness and non-invasive ventilation in case…

The Muscular Dystrophy Association (MDA), the world’s leading nonprofit health agency dedicated to find treatments and cures for muscular dystrophy and other neuromuscular diseases recently announced that it will rally along with the Duchenne muscular dystrophy (DMD) community on the World Duchenne Awareness Day. For the first time the…

aTyr Pharma, Inc. a company engaged in the discovery and development of Physiocrine-based therapeutics to address severe rare diseases, recently announced it began dosing the first patient in a new long term safety extension trial of Resolaris for adult patients suffering from facioscapulohumeral muscular dystrophy (FSHD), a rare and severe genetic myopathy…