Ultragenyx Pharmaceutical, Kyowa Kirin bag Crysvita FDA approval for TIO

Ultragenyx Pharmaceutical, Kyowa Kirin bag Crysvita FDA approval for TIO

Crysvita FDA approval for TIO : Ultragenyx Pharmaceutical and Kyowa Kirin have secured approval for their recombinant fully human monoclonal IgG1 antibody Crysvita (burosumab) from the US Food and Drug Administration (FDA) for the treatment of tumor-induced osteomalacia (TIO). The FDA approval of Crysvita is for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia […]

KIK-AS clinical trial : Angelman syndrome drug candidate GTX-102 to be evaluated by GeneTx Biotherapeutics

KIK-AS clinical trial : Angelman syndrome drug candidate GTX-102 to be evaluated by GeneTx Biotherapeutics

GeneTx Biotherapeutics is set to start the KIK-AS clinical trial for evaluating GTX-102 for the treatment of Angelman syndrome (AS), a rare, neurogenetic disorder resulting from the loss-of-function of the maternally inherited allele of the UBE3A gene. In this connection, the biotech startup has secured institutional review board (IRB) approval from Rush University Medical Center […]