Karen Guillmeno’s son, Hunter, was diagnosed with Duchenne muscular dystrophy — a rare genetic disorder involving progressive muscle degeneration — in 2016. The condition primarily affects males in ...

Duchenne muscular dystrophy (DMD) is a genetic disease in which muscle cells fail to produce a protein called dystrophin. Unlike some other forms of muscular dystrophy, in Duchenne there is a complete ...

Involvement of different brain dystrophin isoforms was assessed in 3 groups of young males diagnosed with DMD. On their first assessment, 43 of the 70 patients with DMD had normal intelligence ...

Werner is CEO of Alltrna and CEO-partner at Flagship Pioneering. Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take) than in the last couple of decades combined. In ...

Duchenne muscular dystrophy (DMD) is a severe genetic condition characterized by progressive muscle weakening. People with DMD typically only live into their 20s, but life expectancy has been ...

LOS ANGELES (KABC) -- A first-of-its-kind gene therapy was just approved to treat Duchenne muscular dystrophy, a devastating condition affecting roughly 1 out of 3,500 to 5,000 boys. The game-changing ...

The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to disease-related loss of muscle and movement, adverse effects of ...

Please provide your email address to receive an email when new articles are posted on . RGX-202 was safe and well-tolerated in all 12 patients at two different doses with no serious adverse events.

Japanese drugmaker Nippon Shinyaku said on Monday that its Duchenne muscular dystrophy drug, Viltepso, part of a controversial class of treatments for the rare disease, had failed to reach its primary ...

This quick alphabetical guide will help you feel more confident when talking about muscular dystrophy with your doctor and loved ones. Muscular dystrophy is the name for a group of genetic diseases ...

A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...