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Common Drug Improves Blood Flow in Muscular Dystrophy

Dramatic effect seen with dose-sensitive response to tadalafil, sildenafil in boys with DMD

Common Drug Improves Blood Flow in Muscular Dystrophy

THURSDAY, May 8, 2014 (HealthDay News) -- Phosphodiesterase type 5 (PDE5) inhibition improves blood flow in the muscles of boys with Duchenne muscular dystrophy (DMD), according to a small study published online May 7 in Neurology.

Michael D. Nelson, Ph.D., from the Cedars-Sinai Medical Center in Los Angeles, and colleagues assessed exercise-induced attenuation of reflex sympathetic vasoconstriction in 10 boys with DMD and 10 healthy age-matched male controls. Simulated orthostatic stress was used to induce reflex vasoconstriction, which was followed by an open-label, dose-escalation, crossover trial with single oral doses of tadalafil or sildenafil.

The researchers found that sympatholysis was impaired in boys with DMD -- producing functional muscle ischemia -- despite contemporary background therapy with corticosteroids alone or in combination with cardioprotective medication. Standard clinical doses of either tadalafil or sildenafil to inhibit PDE5 alleviated this ischemia in a dose-dependent manner. The exercise-induced increase in skeletal muscle blood flow was normalized with PDE5 inhibition.

"These data provide in-human proof of concept for PDE5 inhibition as a putative new therapeutic strategy for DMD," the authors write.

Several authors disclosed financial ties to pharmaceutical companies, including Eli Lilly, the manufacturer of tadalafil.

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