Dr. Lorna Speid, President of PRDPF! spoke at the American ASCPT Meeting in 2016. Her presentation has been published in the ASCPT’s peer reviewed journal. You can find the reference below. In the publication, Dr. Speid provides a summary of the challenges faced by patients with rare diseases.
Speid, L. (2016), Don’t Do Different Things – Do Things Differently! Drug Development in Rare Diseases: The Patient’s Perspective. Clin. Pharmacol. Ther., 100: 336–338. doi:10.1002/cpt.403.”