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Pilot Trials Now Donations

Action Duchenne has already committed £175K for this project we need to raise another £175K to fund further trials.

All donations to this page will go directly to funding the Pilot Trials Now programme. Action Duchenne guarantees that 100% of your donation will go towards funding clinical trials for Duchenne within the PTN programme.
Contact nick@actionduchene.org or phone 0208 556 9955 for details of the programme. Progress reports will be posted on our website and at our annual conference.

PTN

Pilot Trials Now is funding clinical trials of existing drugs for other diseases that might be of benefit to boys living with Duchenne. PTN is an international partnership between leading Duchenne non profits.

We have already begun preparing for the first clinical trial supported by Pilot Trials Now. Pilot Trials Now will give boys with DMD a fighting chance until a cure is found.

Pilot Trials Now partners with carefully selected physicians and fully supports them as they conduct clinical trials of FDA-approved drugs that show promise for DMD. For our first trial, Prinicpal Investigator Kathryn Wagner, MD, PhD will lead a team of five experienced physician-researchers at Kennedy Krieger Institute of Johns Hopkins University. The team will test a drug called sildenafil (commonly known as Viagra). Originally developed by Pfizer as a heart medication, sildenafil has been shown to delay and even prevent heart failure in the DMD animal model. In a study supported by Charley’s Fund and the Nash Avery Foundation, sildenafil significantly improved the cardiac and vascular function of treated mice, and permitted increased blood flow to skeletal muscles. Heart failure is a leading cause of morbidity in DMD patients. Thirty boys ages 16+ will participate in the year-long trial, which will begin in 2010. Pilot Trials Now partners with carefully selected physicians and fully supports them as they conduct clinical trials of FDA-approved drugs that show promise for DMD.

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