NEWPORT BEACH, California, August 10, 2014 Ì¢âÂÛÏ A new Ì¢âÂÒroute forward for therapyÌ¢âÂÌ_ for patients with Duchenne muscular dystrophy, a degenerative disease that mostly afflicts young boys, has been reported by Dr. Kevin Flanigan, principal investigator at the Center for Gene Therapy at Nationwide ChildrenÌ¢âÂã¢s Hospital in Columbus, Ohio.
CureDuchenne is pleased to share the following information from Sarepta Therapeutics. They provided a summary of the regulatory developments and clinical updates. Sarepta is dedicated to helping DMD patients and […]
Ryan Getzlaf, captain of the Anaheim Ducks, is hosting the fourth annual Getzlaf Golf Shootout to benefit CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy.
Today marks a much anticipated major milestone in the treatment of Duchenne, as Translarnaâ„¢ (ataluren) was granted conditional marketing authorization in the European Union for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older.
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by mutations in the dystrophin gene, without curative treatment yet available.
We are on Day 17 of the 79 Days of Duchenne. We are amazed by all the talented and caring people who have participated in the 79 Days of Duchenne. Thank you […]
FasterCures features a Q&A with Debra Miller, founder and CEO of CureDuchenne, in their Innovator Spotlight. FasterCures, a center of the Milken Institute, is an action tank driven by a […]
The transplantation of myoblasts obtained from a healthy donor is a potential treatment of Duchenne muscular dystrophy (DMD). Following intramuscular injection, donor myoblasts can fuse with the myofibers of the patient and introduce the normal dystrophin gene. In a previous Phase 1A clinical trial, (Dystrophin expression in muscles of DMD patients after high-density injections of normal myogenic cells, https://www.ncbi.nlm.nih.gov/pubmed/16691118?dopt=Abstract) the investigators showed that transplantation of myoblasts grown from the muscle biopsy of a healthy donor introduced the normal dystrophin gene in the DMD myofibers, with the consequent expression of the normal dystrophin mRNA and restoration of the dystrophin protein in several myofibers.
Duchenne families: If you have Duchenne muscular dystrophy and are between the ages 18-25 years old, or if you have a son with Duchenne that age, you can help […]
