Phase III trial restarts in race to develop DMD gene therapy

29 April 2022

People with Duchenne muscular dystrophy (DMD) have been given renewed hope that an investigational gene therapy under development by Pfizer (NYSE: PFE) may yield results.

The New York-based company has restarted testing of fordadistrogene movaparvovec after a clinical hold on the candidate was lifted by the US Food and Drug Administration.

Consequently, Pfizer said it would open the first US sites in the Phase III CIFFREO study, which will evaluate the impact of the mini-dystrophin gene therapy on ambulatory patients with DMD.

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