Genetic mutations which occur naturally during the earliest stages of an embryo's development can cause the severe birth defect spina bifida, University College London scientists suggest, in Nature Communications.
PTC Therapeutics Inc is now accepting applications for the 2021 STRIVE Awards Program for Duchenne muscular dystrophy. The deadline to apply is March 15. Winners will be announced in September 2021.
Caring for patients with physical disabilities and debilitating health conditions involves not only providing the means to help them improve their functional abilities, but also being a partner in helping them live their lives on their terms. Jean L. Minkel, PT, ATP shares her experience in this feature.
Duchenne UK, the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) announce a joint grant of $686,500 to Dr William Evans (principal investigators) at the University of California, Berkeley to use a novel method to measure changes in total muscle mass in Duchenne muscular dystrophy patients through biomarkers in urine samples.
Read More
Parent Project Muscular Dystrophy (PPMD)’s Newborn Screening Pilot has been successfully screening babies born in New York State for Duchenne for over a year. Data were presented at the Association of Public Health Laboratories Newborn Screening Virtual Symposium about the ongoing pilot in New York State.
Read More
The Muscular Dystrophy Association (MDA) has elected Donald S. Wood, PhD, as President and Chief Executive Officer, it announces. Dr Wood previously was Vice Chairman of the Board for the MDA and Vice President of Institutional Effectiveness at Odessa College.
Read More
The FSHD Society releases Voice of the Patient Report, a publication based on its June 29, 2020, externally led patient-focused drug development (EL-PFDD) meeting.
Read More
CureDuchenne Ventures announces an investment in Mesentech Inc, a regenerative medicine company with a prodrug platform that selectively delivers therapeutics to bone. The investment is part of a new joint funding collaboration with the Charles H. Hood Foundation (CHF) that looks to advance early-stage research for pediatric conditions.
Read More
A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), UT Southwestern researchers suggest, in the Journal of the American Heart Association.
Read More
The Muscular Dystrophy Association (MDA) announces the launch of the neuroMuscular ObserVational Research (MOVR) Visualization and Reporting Platform (VRP) as a way to help advance neuromuscular disease research and improve patient care.
Read More
The Muscular Dystrophy Association (MDA) has added Garcelle Beauvais, Aloe Blacc, Bryan Cranston, Adam Devine, Fat Joe, Liz Gillies, Aldis Hodge, Edwin Hodge, DJ Khaled, Daniel Levy, Loni Love, Leslie Mann, Robin Thicke, Gabrielle Union Wade and John David Washington to the lineup for The MDA Kevin Hart Kids Telethon, it announces.
Read More
Pfizer Inc’s investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA), it announces.
Read More
LGMD Awareness Foundation Inc announces its formation as a 501(c)(3) non-profit advocacy organization dedicated to advocating for and raising awareness of Limb Girdle Muscular Dystrophy (LGMD) around the world.
Read More
The Muscular Dystrophy Association (MDA) announces MDA Let’s Play For A Cure, a multi-week streaming event featuring 7 weeks of live gaming and esports events produced and livestreamed by MDA.
Read More
The Muscular Dystrophy Association (MDA) announces the awarding of 15 new MDA grants totaling more than $4 million toward research focused on a variety of neuromuscular diseases.
Read More
By targeting MKP5, an enzyme that had been considered “undruggable,” researchers at Yale University have identified a possible treatment path for Duchenne muscular dystrophy.
Read More
The Muscular Dystrophy Association (MDA) announces the relaunch of its Jerry Lewis MDA Telethon with Kevin Hart joining as host. The MDA Kevin Hart Kids Telethon will be a 2-hour special airing globally on Saturday, October 24, 2020 at 8:00pm ET (streaming partners to be announced).
Read More
PTC Therapeutics Inc announces the winners of its sixth annual STRIVE Awards grant program for Duchenne muscular dystrophy.
Read More