by CheckRare Staff | Jan 20, 2022
Ajai Chari, MD from the Mount Sinai School of Medicine discusses the TRiMM-2 study of teclistamab and talquetamab in combination with daratumumab in the treatment of relapsed/refractory multiple myeloma. Results from this study were recently presented in two...
by CheckRare Staff | Jan 17, 2022
Bradley Galer, MD, Executive Vice President and Chief Medical Officer, Zogenix, discusses MT1621, an investigational treatment for thymidine kinase 2 deficiency (TK2d). TK2d is an ultra-rare genetic mitochondrial disease characterized by progressive myopathy,...
by CheckRare Staff | Jan 11, 2022
Sihoun Hahn, MD, Director of the Wilson Disease Center of Excellence at Seattle Children’s Research Institute, discusses the diagnosis and management of Wilson disease. Wilson disease is a genetic disorder caused by mutations on the ATP7B gene. These mutations...
by CheckRare Staff | Jan 11, 2022
Monica Fay, PharmD, Senior Vice President of Global Medical Affairs at Apellis Pharmaceuticals, discusses recent data from the PRINCE study, a phase 3 study of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare,...
by CheckRare Staff | Jan 1, 2022
A group of leading experts in pediatric neurology and movement disorders attended a virtual roundtable to discuss diagnostic, symptomatic, and research aspects of aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is characterized by a...
