An experimental gene therapy for Duchenne Muscular Dystrophy (DMD) has showed better-than-expected results in a three-patient trial, according to preliminary data. Company shares jumped 60 percent ...

Without functional dystrophin to support muscle strength and stability, muscle fibers are easily damaged. Duchenne muscular dystrophy results from mutations in the DMD gene that encodes the dystrophin ...

Gene therapies have set a high bar for outcomes in pharma by delivering curative treatments for difficult diseases in a one-and-done punch. But for Duchenne muscular dystrophy, the results of ...

In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne muscular dystrophy. In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne ...

People with DMD are missing muscle-protecting protein. This is due to a fault in the gene producing dystrophin. Muscles to become weaker over time until it affects the whole body. It is caused by ...

DMD is a severe genetic disorder characterised by weakness and gradual muscle degeneration. Pfizer Duchenne muscular dystrophy development head Dan Levy said: “We are extremely disappointed that ...