Parents To Seek FDA Orphan Drug Designation For Cyclodextrin To Treat Rare and Fatal Cholesterol Disease

On Tuesday morning at 11am, Addi and Cassi are going to undergo volumetric CT scans of their livers and spleens.  The tests are being conducted by doctors to determine if the cyclodextrin IV infusion treatment the twins are receiving under compassionate use INDs approved by the FDA are reducing the size of their enlarged cholesterol […]

FDA Investigational New Drug Application Documents for Cyclodextrin Treatment for Niemann Pick Type C Disease

Here you will find detailed documents outlining Addi and Cassi’s cyclodextrin infusion treatments. We filed a compassionate use IND application to the US FDA in early 2009 to give intravenous cyclodextrin treatment which was approved.  In September 2010, FDA approved a new request to put HPBCD directly into the brains of the twins.  Addi and […]

University of Notre Dame Airs Video During Football Game Highlighting Rare Cholesterol Disease

Thanks to the University of Notre Dame for highlighting the Parseghian Family’s fight against Niemann Pick Type C disease during the Notre Dame versus University of Nevada Reno football game.   They have a true commitment to helping fight not only Niemann Pick Type C but rare disease of all types. A number of people […]

Want to Avoid Alzheimer’s? Keep Cholesterol levels low a new Kaiser Permanente study reports

I have been saying this for close to two years now about cholesterol and dementia being linked but no one seems to listen.  Now a Kaiser Permanente study published in Dementia and Geriatric Cognitive Disorders has hits the news showing that what’s good for your heart is good for your brain.  Really?  I guess this […]

CNN Covers How California Health Care Budget Cuts Threaten Life of Little Girl Suffering From Deadly Cholesterol Disease

Embedded video from CNN Video Here is a story on sweet little Jessica Leoni who suffers from the same disease as Addi and Cassi. Her health care is being threatened by California’s Health Care crisis. Here is the story from CNN. LOS ANGELES, California (CNN) — Anthony and Lisa Leoni have little time to worry […]

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