A new gene therapy treatment for Duchenne muscular dystrophy show promise of arresting the decline of the muscles of those affected by this inherited genetic disease, and perhaps, in the future, repairing those muscles. The laboratory results not only show promise to treat patients with this severely debilitating and incurable…

Tevard Biosciences has announced a four-year collaboration with Vertex Pharmaceuticals to develop new tRNA-based therapies for patients with Duchenne muscular dystrophy (DMD) caused by nonsense mutations. The deal includes options to expand into additional muscular dystrophies and a second indication. “Given Vertex’s proven track record of developing novel therapies to…

A research team led by the University of Portsmouth in the U.K. has discovered that the Duchenne muscular dystrophy gene DMD is expressed differently in a range of different cancers in addition to causing the inherited degenerative muscle disease. As reported in the journal Cancers, they found that many cancerous…

REGENXBIO is launching a Phase I/II trial of its gene therapy, RGX-202, for Duchenne muscular dystrophy. RGX-202 delivers a transgene for a novel microdystrophin protein that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 is designed for delivery and targeted expression of genes…

A new trial may have pinpointed the first treatment that provides meaningful functional improvements in the most advanced cases of Duchenne muscular dystrophy (DMD). Researchers at UC Davis Health and six other sites showed that a cellular therapy appears to be safe and effective in stopping the deterioration of upper…

A team of scientists at Children’s National Hospital, Washington, DC, has adopted a new approach to develop a gene therapy for limb-girdle muscular dystrophy 2B (LGMD2B) that circumvents the need for targeting hard-to-reach degenerating muscles with large doses and packaging the large dysferlin gene into a carrier vector—the two primary…

The Phase Ib trial assessing Pfizer’s mini-dystrophin gene therapy candidate PF-06939926 in Duchenne muscular dystrophy (DMD) was placed on hold by the FDA after the company acknowledged the death of a young male participant in the open-label study. The study was designed to assess a single intravenous infusion of PF-06939926…

Sarepta Therapeutics has announced the initiation of a new global study of SRP-9001 for the treatment of Duchenne Muscular Dystrophy (DMD). The study, known as EMBARK, will be carried out in partnership with Roche. SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue…

The genetic disorder Duchenne muscular dystrophy (DMD) is characterized by progressive muscle degeneration and weakness resulting from alterations of the protein dystrophin that helps keep muscle cells intact. Until recently, boys with DMD usually did not survive much beyond their teen years, but advances in cardiac and respiratory care have…

A gene mutation in the cystic fibrosis gene may explain why some people with Duchenne Muscular Dystrophy (DMD) experience cardiac problems at an earlier stage to others according to research from the University of Texas Southwestern Medical Center, which could help clinicians to better personalize treatment for these patients. DMD…

Researchers at UPenn have found a way to modify gene expression without modifying genome coding, opening the door for future possibilities of treatment for patients with Duchenne muscular dystrophy (DMD). The team of researchers have identified a group of small molecules that may aid in the  development of new therapies…

CRISPR-Cas9 has, for the first time, been tested by systemic delivery in a large animal—and the results are striking. Working in a dog model of Duchenne muscular dystrophy (DMD), the gene editing not only restored the expression of the protein dystrophin, it also improved muscle histology in the dogs. Eric…

Scientists have developed a nonviral approach to delivering the CRISPR/Cas9 gene-editing system to cells that when tested in the mdx mouse model of Duchenne muscular dystrophy (DMD) repaired the faulty DMD gene, leading to improved strength and agility and reduced fibrosis. The platform, called CRISPR-Gold, uses gold nanoparticles to encapsulate…

Gene therapy for conditions such as muscular dystrophy has remained elusive because the large size of the gene prevents them from being packaged on the delivery vehicle used for most successful gene therapies—adeno-associated viruses (AAVs). But now, a team of researchers at the University of Rochester say they have developed…

Sarepta is shining, as its Duchenne Muscular Dystrophy (DMD) gene therapy Elevidys has been granted the much wider label the biotech was aiming for—making it available to four-year-olds and older. This news comes just about a week after Pfizer, which was fast on Sarepta’s heels with their own gene therapy…