Top Hospitals and Doctors Fail To Find Elevated Bilirubin Levels and Enlarged Spleen at Birth as Niemann Pick Type C disease

Chase is fighting for his life and battling against a fatal neurodegenerative disorder called Niemann Pick Type C

Chase was born on August 21, 2009, in Jacksonville Florida. He arrived a bit early via C-Section. At two days old, his bilirubin levels were elevated and a roller-coaster ride to diagnosis began for our family.  At first, Chase was misdiagnosed with Cholestatic Liver Disease […]

WSJ Health Blog Reports – Results in Mice May Help Shape Clinical Trial for Children With Rare Fatal Disease

By Amy Dockser Marcus, Wall Street Journal Health Blog It’s a well-known maxim in science that curing a disease in a rodent doesn’t guarantee the same results in a human being. But new research on using the drug cyclodextrin in mice is likely to help […]

PARENT DRIVEN RESEARCH – Innovations from the trenches!

A Push to spur more drugs for deadly rare diseases LAURAN NEERGAARD, AP Medical Writer Cassidy Hempel, 6, waves at hospital staff with the help of her mother Chris at the Children’s Hospital and Research Center in Oakland, Calif., Friday, March 18, 2011. Cassidy and […]

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. – […]

A Mom Brokers Treatment For Her Twins’ Rare and Fatal Illness

Easter-with-Girls

Bucking Scientific Convention, Ms. Hempel Gets Researchers From Different Fields to Share Data on Potential Therapy By AMY DOCKSER MARCUS – April 3, 2009 From the moment her twin daughters, Addison and Cassidy, were diagnosed with a fatal genetic disease in October 2007, Chris Hempel […]

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.”  […]