News

The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug application for a micro-dystrophin gene therapy program by Sarepta Therapeutics and Nationwide Children’s Hospital, in clinical development for Duchenne muscular dystrophy (DMD). A Phase 1/2a clinical trial is now enrolling participants and should begin dosing…

The Hope for Gus Foundation and its signature Everest to End Duchenne fundraising trek to Mount Everest Base Camp have raised $100,000 to fund new gene-editing research that could lead to new treatments for Duchenne muscular dystrophy (DMD). The nine-member U.S. Everest to End Duchenne team…

The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for the GALGT2 gene therapy program for Duchenne muscular dystrophy (DMD), Sarepta Therapeutics and Nationwide Children’s Hospital recently announced. Following the FDA clearance, Nationwide Children’s will continue to advance the program by initiating a…

A new method using DNA isolated from a blood sample was far more sensitive in detecting large mutations causing Duchenne muscular dystrophy than currently used genetic diagnostics tools, according to a new study. The next-generation mapping method, using the Bionano Genomics platform, has the potential to replace less sensitive…

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…

Sarepta Therapeutics is working with Duke University’s biomedical engineering program program on gene editing therapies for Duchenne muscular dystrophy. The editing approach the partners are using is called CRISPR/Cas9. It can correct mutations by removing regions of a gene that contain abnormal material. Removing the region restores the function of the protein that…

Infection by the influenza virus, commonly known as the flu, was found to contribute to muscle degeneration in a zebrafish model of Duchenne muscular dystrophy (DMD). Scientists often use zebrafish in research due to their genetic structure, which is similar to that of humans. The findings were reported in the…

The nonprofit CureDuchenne launched a new resource for the Duchenne muscular dystrophy (DMD) community called the CureDuchenne Cares Virtual Durable Medical Equipment (DME) Expo. CureDuchenne was founded in 2003 to help save the lives of those with DMD. Through CureDuchenne Cares, the organization has brought innovative…

Results from the HOPE-1 trial of Capricor Therapeutics’ cell-based therapy CAP-1002 to treat boys and men at advanced stages of Duchenne muscular dystrophy (DMD) will be released at a late-breaking session at the American Heart Association Scientific Sessions 2017. In a session…

The U.S. Food and Drug Administration decided not to approve Translarna (ataluren), by PTC Therapeutics, as a treatment for specific types of Duchenne muscular dystrophy caused by “nonsense” mutations. Although not entirely unexpected, the decision was disappointing to many in the Duchenne community. On Sept. 28, an FDA advisory…