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Miglustat (Brazaves)

Niemann-Pick disease type C

ApprovedCommercial

Key Facts

Indication
Niemann-Pick disease type C
Phase
Approved
Status
Commercial
Company

About Nippon Shinyaku

Nippon Shinyaku is a well-established, revenue-generating Japanese pharmaceutical company with a strategic focus on creating novel treatments for unmet medical needs. Its commercial success is anchored by Viltepso, an exon-skipping therapy for Duchenne muscular dystrophy, alongside other marketed products. The company leverages its research expertise to advance a pipeline spanning rare genetic diseases, oncology, and metabolic conditions, aiming for sustainable growth through both internal R&D and strategic partnerships.

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