• PBD-001
    Keywords: primary tetrahydrobiopterin deficiency (PBD), recessive guanosine triphosphate cyclohydrolase 1 (GTP-CH) deficiency, 6-pyruvoyl-tetrahydropterin synthase (PTPS) , CNSA-001, Censa, sepiapterin
    Principal Investigator: Nicola Longo
    Status: Not yet recruiting