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...
by Peter Ciszewski | Mar 7, 2022
Raymond Wang, MD, Metabolic Specialist and Director of the Multidisciplinary Lysosomal Storage Disorder Program at Children’s Hospital of Orange County, discusses RGX-111, an investigational gene therapy for mucopolysaccharidosis type I (MPS I). Data from...
by CheckRare Staff | Mar 6, 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. Dr. Pearl...
by Peter Ciszewski | Mar 5, 2022
Steve Pakola, MD, Chief Medical Officer for Regenxbio, discusses data from the ongoing gene therapy trials in children with mucopolysaccharidosis type I (MPS I) and mucopolysaccharidosis type II (MPS II). The data was presented at WORLDSymposium 2022. MPS I is...
