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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  

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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 …