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...
by CheckRare Staff | Mar 10, 2022
« Prev1 / 1Next »AADC Deficiency Panel DiscussionAADC Deficiency Recognition and CharacterizationAADC Deficiency: Screening and DiagnosisAADC Deficiency TreatmentAADC Deficiency: Patients in the Real World Setting« Prev1 / 1Next » Panel...
by CheckRare Staff | Mar 8, 2022
Karuppiah Kannan, Senior Director – Global Program Leader at Takeda Pharmaceuticals, discusses early results of a phase 1/2 study evaluating subasumstat (TAK-981) in combination with rituximab in multiple subsets of CD20-positive relapsed/refractory non-Hodgkin...
