by CheckRare Staff | Apr 14, 2022
Terence R. Flotte, MD, Provost and Executive Deputy Chancellor of the University of Massachusetts Medical School, gives an overview of Tay-Sachs disease. As Dr. Flotte explains, Tay-Sachs disease (GM2 Gangliosidosis) is a rare neurodegenerative disease caused...
by CheckRare Staff | Apr 8, 2022
Nicole Casale (President) and Brittany Cudzilo (Vice President) of the Galactosemia Foundation are both mothers of children with Type 1 galactosemia. In this video, they describe the mission and goal of the Foundation: (1) to educate, support, and provide...
by CheckRare Staff | Apr 2, 2022
The U.S. Food and Drug Administration (FDA) has approved axicabtagene ciloleucel (Yescarta), a CAR T-cell therapy, for the treatment of adult patients with relapsed or refractory large B-cell lymphoma. Large B-cell lymphoma is a rare cancer and the most common...
by CheckRare Staff | Mar 29, 2022
Caroline Hastings, MD, Hematologist-Oncologist and Neuro-Oncologist from the UCSF Benioff Children’s Hospital, discusses some the ‘tell-tale’ signs of children with Niemann-Pick disease Type C (NPC). NPC is a disabling neurogenetic disorder that has been...
by CheckRare Staff | Mar 23, 2022
Mathias Schmidt, PhD, President and CEO of JCR Pharmaceuticals USA, discusses long-term efficacy and safety data of pabinafusp-alfa (Izcargo) in mucopolysaccharidosis type II (MPS II; Hunter syndrome). Data from this study was recently presented at...
