Progressing the Science in Duchenne Muscular Dystrophy

Progressing the Science in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare, serious, debilitating childhood genetic disease characterized by progressive muscle degeneration that leads to injury and weakness, and a significantly shortened life expectancy. DMD is the most common form of muscular dystrophy worldwide and primarily affects boys.

There is an urgent need to advance DMD research because there are limited treatment options available. As a result, DMD is a focus area of clinical research for Pfizer Rare Disease. The first patients in our Phase 1b clinical trial for our mini-dystrophin gene therapy candidate, PF-06939926, have received an infusion of the mini-dystrophin gene, and enrollment as well as dosing continues. In addition, Pfizer has entered into a research collaboration with the biotechnology company CYTOO to modify their existing MyoScreen™ platform to enable its potential use as a DMD target discovery platform. We have also joined forces with the charity Duchenne U.K. to pioneer a novel approach to Health Technology Assessment.