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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Niemann-Pick Type C News
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 …
Studying Rare Diseases Can Yield Advances For All Of Us
August 11, 2019 • By Elsa Partan & Heather Goldstone • Living Lab Radio on WCAI-FM, Cape Cod, MA Listen to the entire radio interview here: It’s tough to study rare diseases. Because they affect only a small percentage of the population, it can be hard for researchers to find funding. It’s also challenging to …