Holly Peay, Vice President, Education & Outreach

We were thrilled that 119 parents/guardians were willing and able to complete the entire treatment preferences survey. We are analyzing the data. We are doing the first part of the data analysis in-house, and the second part with our health economist collaborator. Our plan is to present this information to the FDA, to industry and clinical…

By now you’ve probably heard about PPMD’s Risk/Benefit in Duchenne Therapies program, which aims to inform the FDA and other government agencies, biopharmaceutical companies, and others about the treatment priorities and risk tolerance of the Duchenne community. We’ve had a great response to our first survey, with more than 90 participants so…

The Duchenne community has entered a new era where more clinical trials are coming online and the possibility of treatment is becoming a reality. Upon completion of a trial, the FDA weighs the evidence presented. But at the end of the day, it is often a value judgment about potential benefits and risks to the patient.

PPMD has launched a program – Benefit/Risk in Duchenne Therapies – to help inform the FDA and other government agencies, biopharmaceutical…

Families of children with muscular dystrophy and other muscle disorders often experience a diagnostic odyssey that is too long and too emotionally difficult. PPMD, with support from The Centers for Disease Control and Prevention (CDC), created the National Task Force for Early Identification of Childhood Neuromuscular Disorders to address the continued gap between the time when the first symptoms of muscle weakness…