Rare Diseases

Erectile dysfunction drugs may be solution for Duchenne muscular dystrophy

Drugs that treat erectile dysfunction may be effective for boys suffering from Duchenne muscular dystrophy (DMD), Medical News Today reported.

Duchenne muscular dystrophy (DMD) affects approximately 1 in every 3,600 male infants worldwide. The inherited condition causes severe muscle weakness and primarily affects boys and young men. DMD can cause intellectual disability, congestive heart failure or irregular heart rhythm, back and chest deformities, and respiratory disorders.

Corticosteroids — which slow down muscle degeneration and reduce DMD’s effects on the heart and lungs — have been used as treatment for the condition, but 75 percent of patients are unable to endure these kinds of drugs.

In order to find an alternative treatment for DMD, researchers studied the effects of two erectile dysfunction drugs, sildenafil (Viagra) and tadalafil (Cialis), on patients with the condition. The drugs help increase blood flow to the muscles by relaxing blood vessels, potentially helping to maintain overall muscle health.

For the study, researchers measured the blood flow of a group of 10 boys between the ages of 8 and 13 who had DMD, as well as a control group of 10 healthy boys. The boys with DMD then took each drug two weeks apart and had their blood flow measured again.

After taking sildenafil and tadalafil, the boys with DMD were revealed to have the same blood flow response as the healthy boys who didn’t take the drugs.

While more research is necessary, the findings are encouraging, researchers noted; however, the effects of long-term use are unknown.

"This proof-of-concept study also does not address the crucial question of whether restoring normal blood flow regulation will preserve muscle and slow disease progression,” said researcher Dr. Ronald G. Victor of Cedars-Sinai Heart Institute in Los Angeles. “If so, this would offer a new therapeutic strategy for Duchenne muscular dystrophy, and a Phase III clinical trial has been launched to find out."

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