by CheckRare Staff | Feb 3, 2022
Philippe Duchateau, PhD, Chief Scientific Officer at Cellectis, describes the company’s TALEN gene editing technology and its application in hematologic cancers. TALEN is based on a class of proteins derived from transcription activator-like effectors (TALEs),...
by CheckRare Staff | Jan 26, 2022
Kathie Bishop, PhD, Chief Scientific Officer at Acadia Pharmaceuticals, discusses the positive top-line results from the pivotal phase 3 Lavender trial testing the efficacy of trofinetide to treat Rett syndrome. Rett syndrome is a rare progressive...
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...
