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 …
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Niemann-Pick Type C 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
Montefiore Einstein Doctor Receives NIH Grant to Expand Newborn Screening and Improve Diagnosis and Access to Treatment
Identifying Rare Diseases In Newborns: Broader Screening For Better Outcomes News provided by Montefiore Health System NEW YORK, Oct. 8, 2019 /PRNewswire/ — Pediatric genetic expert Dr. Melissa Wasserstein has been awarded $3.2 million from the National Institutes of Health (NIH) to conduct the most expansive consented pilot newborn screening study in the country, known …