Jesse's Journey Foundation

Defeat Duchenne. Change Lives.

Pfizer: Update on Phase 1B Open-Label Mini-Dystrophin Gene Therapy Trial

Over the holidays we received some tragic news. We are deeply saddened to learn that a young man participating in the non-ambulatory cohort of Pfizer’s Phase 1b mini-dystrophin gene therapy trial for Duchenne muscular dystrophy has passed away. It is very difficult to find the right words to express how deeply this has touched our community.

Pfizer issued a community letter informing the international Duchenne community of this tragic news:

There is much to learn about what has happened, and as a community, we will come together to get through this.

We would like to re-share the message from Pat Furlong, President & CEO of Parent Project Muscular Dystrophy (PPMD) in the US, as she has so eloquently captured the impact of this devastating news:

If you would like to talk to someone at Jesse’s Journey regarding this upsetting news, please reach out to:

Nicola Worsfold

Director of Research and Advocacy

[email protected]

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