Click on Image to Read Full Story
Download a PDF File
Infusions of Non-Toxic Sugar Compound To Begin Within A Few Weeks At Renown Regional Medical Center
It’s official! The sugar angels have delivered something very sweet to our family. The FDA has approved our "compassionate use" request to give Addi and Cassi infusions of cyclodextrin to see if it can help treat their fatal cholesterol metabolism disease called Niemann Pick Type C. To read more about this novel treatment, see our extensive blog posts on cyclodextrin.
A staggering 60 million people in the US and European Union are impacted by rare diseases of different types. Most people know someone with a "rare disease" or are a caregiver of someone with a rare disease. Please help spread our message to the world — World Rare Disease Day is being held Feb. 28, 2009. For more on how to join the global fight go to
The Project Charity - The Children’s Rare Disease Network.
Addi & Cassi Featured On November 2008 Cover
In the November 2008 issue of The Scientist, author Alison McCook takes and in-depth look at Niemann Pick Type C disease, commonly referred to as the "Childhood Alzheimer’s" and explores the possibility that NPC research could provide insights into the mechanisms of Alzheimer’s disease. Read the entire story here.
Read The Wall Street Journal feature article about CollabRx, a Silicon Valley start-up company we are working with to create one of the first "virtual biotechnology" companies in the United States. CollabRx is helping us develop a therapy pipeline of drugs to treat Niemann Pick Type C kids. We are focused on developing a "combination therapy" of approved drugs and compounds that could prevent or significantly delay the onset and progression of NPC which includes dementia, neuroinflammation and brain tangles.
What Will Obama Do Now?
ReThink IT is an awareness video with an important message for our President elect, Barack Obama. We hope our YouTube video will help raise awareness of the national crisis we are facing as a result of millions of American’s developing Alzheimer’s, dementia and memory disorders. We must all unite NOW to save Medicare from bankruptcy.
