2022 Orphan Drugs: PDUFA Dates and FDA Approvals

Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all...

PIK3CA-Related Overgrowth Syndrome (PROS) Explained

  Guillaume Canaud, MD, PhD, of Necker-Enfants Malades Hospital – AP-HP, the Paris Descartes University, provides an overview of PIK3CA-related overgrowth syndromes (PROS), a group of rare disorders caused by PIK3CA gene mutation. PROS is a group of rare...