申请人:BioMarin Pharmaceutical Inc.
公开号:EP3138566A1
公开(公告)日:2017-03-08
The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.
本发明涉及一种用于治疗干预高苯丙氨酸血症的新型方法和组合物。更具体地说,本说明书描述了使用包含 BH4 的组合物治疗各种类型苯丙酮尿症的方法和组合物。还考虑了 BH4 和其他治疗方案的组合疗法。