Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy

As a community, we are crushed to hear the news from Pfizer that their Phase 3 mini-dystrophin gene therapy trial in Duchenne failed to meet its primary endpoint.  Individuals, aged 4 to 7 years old,  treated with the experimental gene therapy did not show significant improvement in motor function, as measured by the North Star Ambulatory Assessment (NSAA) and compared to placebo, at one year after treatment.  You can read more at the company’s press release HERE.

We know that this news is devastating to the Duchenne community, which has been closely watching this news with great hope. We stand with you in this difficult time as we face this news and move forward together.

We thank Pfizer for their efforts to bring treatments to the community that so greatly needs them. We are immensely grateful to the brave individuals with Duchenne and their families who have participated in clinical trials, advancing research and bringing us closer to transformative treatments. 

CureDuchenne remains unwavering in our mission to find a cure for Duchenne, and we won’t be deterred from that goal.  We know that our work is far from done to get more treatments through the FDA approval process and to individuals with Duchenne who need them so desperately. For this reason, we will continue to support and invest in a diverse array of promising research to bring solutions to everyone living with Duchenne. 

See the letter to the community: