ZY19489 is a novel anti-malarial compound active against all current clinical strains of P. falciparum and P. vivax, including drug resistant strains
Tezepelumab has been granted Orphan Drug Designation (ODD) in the US by the U.S. Food and Drug Administration (FDA) for the treatment of eosinophilic esophagitis (EoE)
Extension of hypericin orphan designation beyond cutaneous T-cell lymphoma
The drug is also intended for the treatment of glioblastoma multiforme
Marks significant milestone for breakthrough biologic to treat rare blood cancers
The therapy, also known as UM171 Cell Therapy, recently received conditional marketing authorization from the European Commission
This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop a therapy for sickle cell disease
The pioneering monoclonal antibody is for Essential Thrombocythemia (ET) and Polycythemia Vera (PV), rare chronic blood cancers with limited treatment options
Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector
The investigational subcutaneous hepcidin mimetic peptide is designed to regulate iron and red blood cell production
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