Treatment found for muscular dystrophy?

• Position: Medication

A drug typically prescribed for erectile dysfunction or pulmonary hypertension restores blood flow to oxygen-starved muscles in patients with a type of muscular dystrophy that affects males, typically starting in childhood or adolescence.

In a clinical trial, tadalafil--commonly known by brand names Cialis and Adcirca--reversed the effects of a biochemical chain of events that, in Becker muscular dystrophy, deprives muscles of an important chemical, nitric oxide, which normally tells blood vessels to relax during exercise, increasing blood flow and oxygenation.

With a single dose, the drug, which works downstream from nitric oxide, fully restored proper blood flow in almost 90% of the patients in the study, and the effects were "both marked and immediate," according to an article in Science Translational Medicine.

"There is no treatment for this progressive muscle-wasting disease and there has been little research. Previous studies suggested that drugs such as tadalafil could restore proper blood flow, but this is the first study showing that the drug may offer a therapeutic strategy," explains cardiologist Ronald G. Victor, senior author.

Becker muscular dystrophy results from a genetic defect that reduces the amount of a protein called dystrophin in the membrane of muscle cells. With insufficient and poorly...