News

UCLA scientists have developed a potential gene therapy approach for Duchenne muscular dystrophy (DMD) using CRISPR/Cas9 technology and stem cells. If the new treatment proves successful and reaches the clinic — possibly in the next decade — it could be applied to 60 percent of all Duchenne patients. CRISPR/Cas9 is a gene-editing technology that allows targeting and…

A discovery clears up the confusion about the molecular factors involved in muscle repair. The research, published in the journal e-life, shows that a protein complex containing the factors TBP and TFIID is essential to activate genes responsible for adult muscle tissue regeneration. Adult muscles contain stem cells that can be activated…

Catabasis Pharmaceuticals announced it will present data on CAT-1004 at the XIV International Conference on Duchenne and Becker Muscular Dystrophy to be held in Rome, Italy, on Feb. 13-14. Joanne Donovan, chief medical officer of Catabasis, will talk about the drug, now in clinical testing, and the design of the MoveDMD…

Sarepta Therapeutics, a developer of RNA-targeted treatments, announced that the U.S. Food and Drug Administration (FDA) is extending by another three months its review of the company’s New Drug Application (NDA) for eteplirsen, a potential treatment for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A reason for the extension was…

Catabasis Pharmaceuticals and the Muscular Dystrophy Association (MDA) have announced a partnership that will support Part B of the MoveDMD clinical trial, currently evaluating the effectiveness and safety of Duchenne muscular dystrophy (DMD) treatment candidate CAT-1004 in children. CAT-1004, believed to be a disease modulator regardless of mutation, is…

In recognition that people with muscular dystrophy are living longer than even recently thought possible, the Muscular Dystrophy Association (MDA) has named a young man as its latest National Goodwill Ambassador — marking the first time in the program’s 64-year history that a child hasn’t been selected for that role.

Jiffy Lube International has raised more than $1 million dollars in a campaign to support the Muscular Dystrophy Association (MDA). The company holds more than 2,000 franchised oil change service centers and has partnered with MDA for the fourth year to support children and adults who suffer from muscular dystrophy,…

The Kessler Foundation and the New Jersey Institute of Technology (NJIT) will share a $5 million federal grant, titled Rehabilitation Engineering Research Center on Wearable Robots, to develop a wearable robotic exoskeleton for use in patients with Duchenne muscular dystrophy (DMD), as well as other applications for spinal cord injury and stroke.

More than a dozen children in the Tampa, Florida area with muscular dystrophy, Down’s syndrome, and other neuromuscular disorders will soon be getting new and specially adapted bicycles donated by the ACMG Foundation for Genetic and Genomic Medicine. The 17 young recipients are from the local chapter of the Muscular Dystrophy Association (MDA) and …

Advances in stem cell therapy using patients’ own cells with genetically modified dystrophin have been hampered by the large size of the dystrophin gene, which are difficult to pack into a delivery system. Now, scientists at University College London have managed to produce a gene construct much bigger than was…