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...
by CheckRare Staff | Mar 15, 2022
Dean Suhr, President and Co-Founder of the MLD Foundation, gives an overview of the treatment landscape for metachromatic leukodystrophy (MLD). MLD is a lysosomal storage disorder caused by arylsulfatase A (ARSA) deficiency. It is characterized by the...
