UF Team Plays Major Role in Success of Newly Approved Duchenne Muscular Dystrophy Drug

Duchenne muscular dystrophy families, advocates and healthcare providers celebrated a milestone with the U.S. Food and Drug Administration’s approval of the first nonsteroidal drug for the treatment of Duchenne. The University of Florida’s expertise in magnetic resonance imaging provided objective, measurable results in the drug’s clinical trials.

In findings published in the April issue of The Lancet Neurology, a team of investigators led by pharmaceutical company Italfarmaco, in collaboration with UF, demonstrated that the drug is associated with slower functional decline and decreased fat replacement in muscle.

“This announcement brings a lot of hope and opportunity to patients with Duchenne muscular dystrophy and their families. It is a very big step forward for the community and provides an effective treatment for families and providers,” said Krista Vandenborne, Ph.D., P.T., a distinguished professor and chair of the department of physical therapy at the UF College of Public Health and Health Professions and a member of the phase 3 clinical trial team that evaluated the new drug, marketed as Duvyzat.

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