DART participated in the town of Greenwich’s second annual, and this year, “virtual” rare disease awareness day. “It was truly and honor for the second year to join other families in Greenwich who are fighting to make a real difference in the lives of those suffering from some of the more than 7,000 rare diseases.” …
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DART 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