News

A gene therapy approach that Benitec Biopharma is developing for oculopharyngeal muscular dystrophy (OPMD) brought impaired muscle strength in mice back to healthy levels and decreased their muscle fibrosis, or scarring, according to a preclinical trial study. Benitec said its approach could become an effective strategy for treating the rare genetic…

Researchers investigated swallowing dysfunctions in Becker muscular dystrophy (BMD) patients and found they have swallowing problems similar to those observed in patients with Duchenne muscular dystrophy (DMD). These observations are important not only for a better understanding of BMD progression, but also to improve the standard care of these patients. The…

Solid Biosciences of Cambridge, Mass., has completed the initial closing of a $50 million Series C round of financing to advance the company’s clinical portfolio of Duchenne muscular dystrophy (DMD) medications and programs. RA Capital Management and Bain Capital Life Sciences are the principal investors, with input also from RTW Investments, Foresite Capital,…

Surgical treatment of severe spine deformities, or scoliosis, can be a treatment possibility for patients with neuromuscular diseases. A case report titled “Spinal fusion in a patient with Fukuyama congenital muscular dystrophy” was published the journal Brain & Development. It showed that a surgical approach for the treatment of progressive scoliosis improved…

The nonprofit CureDuchenne organization has changed its website to better reflect its mission of improving the lives of people with Duchenne muscular dystrophy (DMD). CureDuchenne and the DMD community have taken heart from two recent Federal Drug Administration approvals. The initial one, in September 2016, was for the first-ever drug…

Hundreds seeking a cure for Duchenne muscular dystrophy will join April 8 at the University of Texas (UT) Golf Club for the Champions to CureDuchenne gala, now in its eighth year. The event, hosted by Quan Cosby, former wide receiver on the UT football team, and presented by…

Summit Therapeutics has confirmed plans for an extension stage of a Phase 2 clinical trial evaluating ezutromid as a treatment for boys with Duchenne muscular dystrophy (DMD). …

Data from RECENSUS, a medical chart review of boys with X-linked myotubular myopathy (XLMTM), demonstrates the significant medical burden for children living with the disease, a well as their families and caregivers. Alan Beggs, PhD, director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital, presented data…

Akashi Therapeutics has received an all-clear signal from the U.S. Food and Drug Administration to resume developing a Duchenne muscular dystrophy (DMD) treatment. The FDA had ordered a hold on the development of delayed-release halofuginone (HT-100) after a clinical-trial participant began experiencing life-threatening adverse effects two weeks after beginning the therapy. HT-100 is…

For years, Lexi Pappas hid the fact she had facioscapulohumeral dystrophy (FSHD) — a hereditary muscle wasting disorder that begins in the face and gradually spreads to the shoulders and upper arms, and sometimes also to the legs. As the disease took its toll on Lexi — causing her…