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.
In a recent study entitled “Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice”, published in the Human Gene Therapy journal, a team of researchers from the Nationwide Children’s Hospital and the Ohio State University used gene therapy…
Cellular models can elucidate molecular mechanisms underlying several diseases, reducing animal usage in biomedical sciences and following the guiding principles for animal testing. Now, researchers at the Karolinska Institute and at the Karolinska University published a study entitled “Primary Murine Myotubes as a Model for Investigating Muscular…
A team of research scientists based at the University of Twente Department Of Biomechanical Engineering in Enschede, The Netherlands, is developing a passive arm support device prototype, dubbed A-gear, that runs along the body from the contact surface between the seat and upper legs via the torso and upper…
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,…
Researchers at the University of California published a study entitled “Human Satellite Cell Transplantation and Regeneration from Diverse Skeletal Muscles” in the journal Stem Cell Reports whereby they reveal isolated human muscle stem cells capable of repairing damaged muscles. One of the major problems of treating…
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…
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