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...
by CheckRare Staff | Mar 22, 2022
As part of the roundtable moderated by Philip Pearl, MD, of Boston Children’s Hospital and Harvard Medical School, the attendees of this virtual discussion focused on key diagnostic aspects of aromatic L-amino acid decarboxylase (AADC) deficiency. The entire...
