February 25, 2015 Niemann Pick Disease type C, or NPC, is a disease most people have never heard of, affecting just one person in 150,000. Yet the disease is a devastating one. Frequently diagnosed in children in their elementary school years, sufferers usually die by the time they’re 20. The disease is sometimes referred to …
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Research News
NIH partners with Vtesse on Cyclodextrin clinical trial!
Thank you to all of DART’s supporters. We have great news! The National Institutes of Health announced last Wednesday, while Andrew was getting his 16th monthly dose of Cyclodextrin (see photo), that the NIH has selected pharma company Vtesse to partner on the further development of treatments for NPC, starting with the continuation of the …
WALL STREET JOURNAL REPORTS ON NEW NP-C DRUG TRIAL AT THE NATIONAL INSTITUTES OF HEALTH
Read about the detailed efforts of the Marellas and other NP-C families to bring the latest and most promising therapy for NP-C, Cyclodextrin, into an FDA approved clinical trial in an amazing, online experience! Trials: A Desperate Fight to Save Kids & Change Science. For six years, Wall Street Journal reporter Amy Dockser Marcus followed …