Walgreen’s Support Allows Our Twins with Niemann Pick Type C To Receive Cyclodextrin Treatments At Home

We are sorry for the lack of posts in recent months! Hugh and I have been extremely busy with rare disease initiatives for Solution Therapeutics, our virtual biotech, as well as helping the Global Genes Project with a number of community based rare disease projects. We would like to report that after three years of […]

Meet The Children Who Are Resistant To Contracting Ebola, HIV-AIDS

My life grows more surreal by the day. Four years ago, I was told that my eight year old identical twins have a fatal genetic cholesterol disease called Niemann Pick Type C (the “Childhood Alzheimer’s”) and they would likely not live to see their 10th birthday. Now I have learned from top virus scientists that […]

Why Washington Should Support ULTRA Act (H.R. 3737)

It’s great to see some representatives in Washington are finally starting to understand the real healthcare issue facing tens of millions of American’s afflicted with rare diseases. You can’t improve health if potential medications remain trapped in research and biotech’s “Valley of Death” and never reach patients. This week, U.S. Representatives Cliff Stearns (R-FL) and […]

From The Valley of Death to The Valley of Cures – NIH TRND Program Supports Multiple Rare Diseases

Thank you Dr. Francis Collins, Dr. Chris Austin, Dr. John McKew and others at the NIH who are all involved in driving the National Institutes of Health’s Therapeutics for Rare and Neglected Diseases (TRND) program forward. TRND has been a huge success since launching five pilot projects last year, including one program that focuses on […]

National Institues of Health Announces Cyclodextrin Clincial Trial For Fatal Cholesterol Disease Called Niemann Pick Type C

Over the past three years, many Niemann Pick Type C families as well as physicians treating NPC patients worldwide have contacted Dr. Caroline Hastings and I regarding Addi and Cassi’s treatments with hydroxypropyl-beta-cyclodextrin. Since sharing our intravenous (IV) and intrathecal (IT) cyclodextrin treatment protocols with NPC families and doctors in the U.S. and other countries, […]

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