Adrabetadex (Cyclodextrin) Clinical Trial. As many of you may know, much to the surprise of the NPC community, Mallinckrodt in January 2021 abandoned the Adrabetadex clinical trials; which Andrew Marella has been a part of for over 7 years. Much of the NPC community’s efforts since that announcement had been focused on what would come …
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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 …
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