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