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
Sugar Molecule Used in Common Food and Household Products Like Febreze® Fabric Refresher Called Hydroxypropyl Beta Cyclodextrin (HPßCD) Will be Delivered into Twins’ Central Nervous System in an Attempt to Stop Neurological Progression of Niemann Pick Type C Disease September 23, 2010 Oakland, Calif. – Children’s Hospital & Research Center Oakland announced today that the [...]
Improve FDA’s Rare Disease Review Process
The Jewish Daily – FORWARD Opinion By Jonathan Jacoby The Jewish community has long been a leader in supporting medical research and education efforts, especially with regard to those diseases that disproportionately afflict people of Ashkenazi Jewish descent. Creating coalitions with other patient advocates in the rare disease community would give American Jews an opportunity [...]
Collaboration is key in rare disease research!
KTVU-TV 2, the leading evening news in the San Francisco Bay Area, today reported on Addi and Cassi Hempel, identical twins affected by a rare disease called Niemann-Pick Type C (NPC). NPC is fatal and degenerative cholesterol disease that is often called the “childhood Alzheimer’s.” What is most interesting about this story is that Dr. [...]