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Niemann-Pick Type C News Research News

Mallinckrodt pulls out of cyclodextrin trial – NPC community will continue to fight for it.

On January 20, 2021, Mallinckrodt Pharmaceuticals called Phil Marella of DART and other NPC community leaders and advised us that they were not going to go forward with an FDA new drug application for Adrabetadex/Cyclodextrin. Of course, this is devastating and frightening news for the Marella family and the more than 80 children in the …

DART News Niemann-Pick Type C News Research News

NPC drug Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease

Wonderful scientific confirmation of the benefit for kids from our NPC community’s research efforts! Andrew has been on this medicine since 2004, and our toolbox is just starting to grow! Read the article: https://medicalxpress.com/news/2020-09-miglustat-swallowing-children-adolescents-niemann-pick.html