公开(公告)号：EP4029519A1

公开(公告)日：2022-07-20

申请号：EP22154751.6

申请日：2008-04-11

申请人： BioMarin Pharmaceutical Inc.

发明人： Oppenheimer, Daniel ,  Dorenbaum, Alejandro ,  OKHAMAFE, Augustus ,  Foehr, Erik ,  Castillo, Sianna ,  Kostel, Paul

IPC分类号： A61K45/06 ,  A61K9/20 ,  A61K9/48 ,  A61K31/519 ,  A61P3/00 ,  A61P3/02 ,  A61P25/16 ,  A61K9/00 ,  A61K9/16 ,  A61P7/06 ,  A61P9/00 ,  A61P9/10 ,  A61P25/00 ,  A61P25/06 ,  A61P25/18 ,  A61P25/24 ,  A61P25/28 ,  A61P43/00 ,  C07D487/04 ,  C07B63/00 ,  G01N30/72

摘要： The present invention is directed to treatment methods of administering tetrahydrobiopterin, including in oral dosage forms, in intravenous formulations, and with food for treating BH4-responsive disorders. Also disclosed are biopterin assays for measuring the amount of biopterin and metabolites of biopterin in a sample.