PPMD is proud to join our friends at Duchenne Parent Project NL in supporting the Duchenne-specific submissions to the Journal of Neuromuscular Diseases exploring preclinical and clinical studies that might not have met expectation.

Of course our number one priority is supporting development of safe and effective approved therapies for our loved ones with Duchenne. But as we all know, the road to drug approval is complex and sometimes disappointing. The Journal of Neuromuscular Diseases, in a special issue led by Dr. Annemieke Aartsma-Rus, has put out a call to analyze well-done research and trials which didn’t reach their predicted outcome so that we can learn from the past to improve research and trials today and look towards the future.

Learning from these ‘Negative Results’ will help avoid unnecessary testing in humans and animals, as well as save money and our most precious resource, time, as we continue the fight to End Duchenne.

Dr. Annemieke Aartsma-Rus provided an excellent overview of the translatability of drug studies from pre-clinical models to humans at PPMD’s Virtual Conference, click here to watch her presentation.