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 To The Finish 2020 Raises $145,000!
Thank You to All Who Walked and Donated! We are so happy to announce we raised $145k for crucial NPC researchTHANK YOU!! We are so overwhelmed by the support for our virtual DART TO THE FINISH charity walk this last week. Thank you to all who participated! Walkers joined us from all across the country. It …
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