CureDuchenne is a nonprofit whose mission is to find a cure for Duchenne muscular dystrophy (DMD), a rare progressive disease characterized by muscular degeneration. It is diagnosed in one individual out of every 5,000 males who are born. Those diagnosed with DMD are often unable to walk, eat by themselves, and even breathe on their own. Many individuals are affected by heart failure.

The organization recently released a webinar which discusses the opening of clinical trial locations in the U.S. for a Phase 3 trial sponsored by Pfizer. Pfizer’s Duchenne program was first acquired from a company called Bamboo Therapeutics, which was funded by CureDuchenne back in 2016.

The Trial & Webinar

This trial is called CIFREO. It is a Phase 3 investigation which will examine the safety and efficacy of a gene therapy treatment for DMD.

The webinar by CureDuchenne included a talk by Beth Belluscio, who is the Global Clinical Lead for Rare Neurological Diseases at Pfizer. She presented insight on the trial design, how they are assessing safety, inclusion criteria, and exclusion criteria.

Additionally, Debra Miller, the Founder of CureDuchenne, offered a Q&A session.

You can watch the full webinar here.

CureDuchenne

CureDuchenne is known to be a leader globally for improving awareness, education, research, care for patients, and more, for DMD. The primary goal of the organization is to uncover a cure for the condition. They are working toward that goal through funding research, promoting early diagnosis, as well as education for the community.

You can read more about the organization and its mission here.

You can read more about the Phase 3 clinical trial here.