Bleeding of unknown cause (BUC) is a diagnosis of exclusion, and it is common for these patients to have congenital platelet function disorders. Whole-exome sequencing may help reach a more accurate diagnosis in these cases.
Takeda Pharmaceutical Co. Ltd. and Protagonist Therapeutics Inc. have inked a global development and commercialization deal worth up to $1.7 billion for Protagonist’s rusfertide for treatment of polycythemia vera (PV), a rare and chronic blood disorder affecting bone marrow.
Takeda Pharmaceutical Co. Ltd. and Protagonist Therapeutics Inc. have inked a global development and commercialization deal worth up to $1.7 billion for Protagonist’s rusfertide for treatment of polycythemia vera (PV), a rare and chronic blood disorder affecting bone marrow.
Newly approved gene therapies targeting sickle cell disease will be the first focus of the U.S. Centers for Medicare & Medicaid Services’ (CMS) Cell and Gene Therapy Access Model, the agency said Jan. 30.
E-Therapeutics plc has offered a pipeline update, following the nomination of novel target genes, which have yielded promising results in preclinical studies.
Though the PDUFA date for its BLA wasn’t until March 30, 2024, Vertex Pharmaceuticals Inc. celebrated the U.S. FDA approval Jan. 16 for Casgevy (exagamglogene autotemcel), expanding use the CRISPR/Cas9 gene-edited cell therapy in patients, 12 and older, with transfusion-dependent beta-thalassemia.
China’s National Medical Products Administration (NMPA) has accepted for review Hutchmed’s NDA for sovleplenib (HMPL-523) for treatment of primary immune thrombocytopenia.
China’s National Medical Products Administration (NMPA) has accepted for review Hutchmed’s NDA for sovleplenib (HMPL-523) for treatment of primary immune thrombocytopenia.
Dysregulation of the kallikrein-kinin system (KKS) is involved in hereditary angioedema (HA) pathophysiology; thus, the inhibition of factor XIIa (FXIIa), a central mediator of angioedema and initiator of KKS, is a therapeutic strategy to prevent and treat HA attacks.