by Peter Ciszewski | Sep 27, 2022
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...
by Peter Ciszewski | Sep 23, 2022
Mark Forman, MD, PhD, Chief Medical Officer at Passage Bio, discusses the global phase 1/2 upliFT-D clinical trial evaluating PBFT02, an adeno-associated virus (AAV) delivery gene therapy for patients with frontotemporal dementia (FTD) with granulin (GRN)...
by Peter Ciszewski | Sep 21, 2022
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...
by Peter Ciszewski | Sep 16, 2022
Guillaume Canaud, MD, PhD, of Necker-Enfants Malades Hospital – AP-HP, the Paris Descartes University, explains why surgical intervention to treat PIK3CA-related overgrowth syndromes (PROS) is not always the best option. PROS is a group of rare congenital...
by Peter Ciszewski | Sep 15, 2022
Ruchira Glaser, MD, MS, Senior Vice President, Therapeutic Area Head of Rare Disease, Autoimmune & Cardiovascular at Moderna, discusses the phase 1/2 Paramount study evaluating investigational mRNA-3927 in participants with propionic acidemia (PA). PA is a rare...