Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy organization, to develop gene therapies for limb-girdle muscular dystrophy type 2A (LGMD2A). LGMD2A, also known as calpainopathy, is the most common subtype of limb girdle muscular dystrophy (LGMD),…
Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 to 6 years old. Findings from the trial were published in the study, “Phase IIa trial…
Obsessive compulsive disorder (OCD), a type of “internalizing disorder,” are evident in children with Duchenne muscular dystrophy, and particularly associated with anxiety and places considerable stress on the patient’s family, a small retrospective study reported. Researchers call attention to the importance of care…
Using the gene editing process CRISPR/Cas9 enabled a marked restoration of dystrophin production in various muscles of dogs with Duchenne muscular dystrophy (DMD). The study with that finding, “Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy,” was published in the journal…
PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) awards, which recognize the efforts of nonprofit organizations working to serve the Duchenne muscular dystrophy community. This year’s winners are patient organizations located in Canada, Argentina, Brazil, Croatia, Hungary, Ireland, Slovenia and Turkey.
Nonprofit Sing Me a Story and Numotion are collaborating to help children with various neuromuscular diseases served by the Muscular Dystrophy Association (MDA) share their unique stories through song. This alliance was supported by the ongoing partnership between MDA and Numotion, a company that provides mobility products and services.
Pfizer is halting the clinical development of domagrozumab (PF-06252616) as treatment for Duchenne muscular dystrophy (DMD) in an ongoing Phase 2 trial. The latest trial data failed to demonstrate domagrozumab’s potential to prevent disease progression or improve patient’s physical capacity, as assessed by mean changes (in seconds) from…
With an emphasis on standards of care, groups such as the Parent Project Muscular Dystrophy and the Jett Foundation are gearing up for Sept. 7, World Duchenne Awareness Day (WDAD) 2018. Organizations are recommending a host of ways to raise awareness for Duchenne muscular dystrophy,…
The Pacific Northwest chapter of the FSH Society is holding its first-ever “Walk & Roll to Cure FSHD” event Sept. 22 in Puyallup, Washington, a small city about 35 miles south of Seattle. FSH Society holds these events around the U.S., with the goal of raising funds for its work…
For both children and adults with Duchenne or Becker muscular dystrophy, having a specially trained dog doesn’t just relieve their loneliness. These service dogs also help patients in dozens of practical ways, doing everything from taking off shoes to picking up toys that fall on the floor. Tali…
Get regular updates to your inbox.