美国食品和药物管理局批准Miplyffa用于治疗尼曼-皮克病C型, FDA approves Miplyffa for Niemann-Pick disease type C, a rare neurological disorder.

林业发展局已核准Miplyffa(arimoclomol)为首次治疗Niemann-Pick疾病C型(NPC),这是一种罕见的遗传疾病,引起神经症状和器官机能失调。 The FDA has approved Miplyffa (arimoclomol) as the first treatment for Niemann-Pick disease, type C (NPC), a rare genetic disorder causing neurological symptoms and organ dysfunction. Miplyffa在与2至19岁的儿童和青年进行的为期12个月的试验中进行了评价,通过减缓疾病发展速度证明了其效力。 Evaluated in a 12-month trial with children and young adults aged 2 to 19, Miplyffa demonstrated efficacy by slowing disease progression. 它应该与MIGLUSTAT一起使用。 It should be used alongside miglustat. 常见副作用包括上呼吸道感染、腹泻和体重下降。 Common side effects include upper respiratory infections, diarrhea, and weight loss.

September 20, 2024

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