June 8, 2017
Hayley Fights Niemann Pick Type C Using Compassionate Use Program and Cyclodextrin
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September 22, 2016
Science Magazine: Rare Diseases and Rare Opportunities
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January 7, 2015
WSJ Reports: Small Biotech Gets Rights to Rare Disease Drug
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August 9, 2012
Top Hospitals and Doctors Fail To Find Elevated Bilirubin Levels and Enlarged Spleen at Birth as Niemann Pick Type C disease
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June 23, 2011
WSJ Health Blog Reports – Results in Mice May Help Shape Clinical Trial for Children With Rare Fatal Disease
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April 25, 2011
PARENT DRIVEN RESEARCH – Innovations from the trenches!
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September 24, 2010
Children’s Hospital Oakland Receives FDA Clearance to Begin World’s First Cyclodextrin Administration Into the Brains of Twins with Rare and Deadly Cholesterol Disease
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April 21, 2009
A Mom Brokers Treatment For Her Twins' Rare and Fatal Illness
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April 3, 2009
Collaboration is key in rare disease research!
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