Blog Archives

Dr. Oz Show features “Baby Alzheimer’s”; Niemann-Pick disease parents, types A and C

On December 27, 2021 the Dr. Oz Show featured two families dealing with “Baby Alzheimer’s;” one with a child with Niemann-Pick disease type A and the other with Niemann-Pick disease type C. Here is the story from the show.

Posted in Niemann-Pick Type C News, Research News Tagged with: , , , , ,

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

Posted in Niemann-Pick Type C News, Research News Tagged with: ,

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  

Posted in DART News, Niemann-Pick Type C News, Research News