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John Snow @johnsnow ha aggiunto una foto in Health
2025-10-08 11:42:24

Advancements in Niemann-Pick Disease Type C Treatments: A Path Toward Better Outcomes

Zavesca (Miglustat): The Current Approved Therapy for Niemann-Pick Disease Type C Niemann-Pick Disease Type C (NPC) is a rare, progressive, and life-threatening lysosomal storage disorder marked by neurological and visceral complications. At present, Zavesca (miglustat), also sold as Brazaves, is the only approved therapy available for Niemann-Pick Type C treatment. Miglustat works by...

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