by Peter Ciszewski | Dec 2, 2022
Steven Pipe, MD, Professor of Pediatrics and Pathology, and Pediatric Medical Director of the Hemophilia and Coagulation Disorders Program at the University of Michigan, discusses the results of the phase III HOPE-B clinical trial that led to the recent FDA...
by Peter Ciszewski | Nov 30, 2022
P.J. Brooks, PhD, Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes the Bespoke Gene Therapy Consortium (BGTC). BGTC program is a private-public initiative that is...
by Peter Ciszewski | Nov 25, 2022
Alan Mendelsohn, MD, Chief Medical Officer at Timber Pharmaceuticals, discusses the mechanism of action of TMB-001, an investigational treatment for congenital ichthyosis. Congenital ichthyosis is a rare genetic skin disorder characterized by dry, thickened,...
by Peter Ciszewski | Nov 22, 2022
Diana Castro, MD, founder of the Neurology Rare Disease Center in Dallas, Texas discusses the most common early symptoms observed in the different types of spinal muscular atrophy (SMA). SMA is a genetic disease that affects motor neurons in the spinal cord and...
by Peter Ciszewski | Nov 18, 2022
Mark Forman, MD, PhD, Chief Medical Officer at Passage Bio, gives an overview of frontotemporal dementia (FTD) with granulin (GRN) mutations. As Dr. Forman explains, FTD is a disorder that affects the frontal and temporal lobes of the brain, areas that control...
