Ultragenyx

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The first sign of a child suffering from Angelman syndrome is a delay in development, such as the inability to sit without support or making incoherent babbling sounds. The hallmarks of the syndrome constitute severe physical...

Cell Medica receives funds for its cell therapy A USD 8.7 million grant to Cell Medica has been given by Cancer Prevention and Research Institute of Texas (CPRIT) to speed up the clinical development of CMD-502 off-the-shelf chimeric antigen receptor-natural killer T cell (CAR-NKT) cell therapy. The program, su...