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公开(公告)号:EP3977999A1
公开(公告)日:2022-04-06
申请号:EP21209606.9
申请日:2004-11-17
IPC分类号: A61K31/519 , A61P3/00
摘要: The present invention is directed to novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes compositions comprising tetrahydrobiopterin (BH4) and the use thereof in methods of treating various types of phenylketonurias. Combination therapies of BH4 and other therapeutic regimens are contemplated.