by CheckRare Staff | Apr 15, 2022
David Weinstein, MD, Senior Vice President of Clinical Development at Passage Bio, discusses why the company’s investigational gene therapy (PBGM01) is being administered through the cisterna magna. GM1 gangliosidosis is an inherited lysosomal storage disorder...
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...
