Solving the Rare Disease Equation - Dr. Alaa Hamed, Global Head of Medical Affairs, Rare Diseases at SanofiNov 30, 2022
New Hope for Patients with Black Bone Disease - Nick Sireau, CEO and Chair of Trustees at the AKU SocietyOct 5, 2022
Making Rare Disease Treatments A Priority - John Crowley, Executive Chairman at Amicus TherapeuticsSep 22, 2022
The Critical Role of Newborn Screening - Rare Mom Alison Breitbarth and Infantile Pompe DiseaseMay 12, 2022
A randomized study of alglucosidase alfa in late-onset Pompe's disease by Robert Leshner, MD, and Paula Clemens, MDMar 10, 2011
