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 […]

From Hospital To Home on Christmas

We started Christmas Eve day in the San Francisco Bay Area at Children’s Hospital Oakland where Dr. Caroline Hastings gave Addi and Cassi their sixth injections of cyclodextrin into their central nervous systems. By 1 pm, Addi and Cassi were feeling a lot better and we raced home on Highway 80 up over Donner Summit, […]

Family of Twins with Rare and Fatal Cholesterol Disease To Seek FDA Approval To Deliver Cyclodextrin Into the Brain

The tagline of the new movie, Extraordinary Measures, about John Crowley’s fight to save his children from the rare and fatal Pompe disease is….Don’t Hope For a Miracle – Make One! We are now working on making our own miracle  – a new compassionate use investigational new drug (IND) protocol and application that we plan […]

FDA Approves First Ever Cyclodextrin Infusion Treatment For Twin Girls Suffering From Fatal Cholesterol Disease

After months of research, document preparation, discussions with the FDA and an immense amount of stress, it’s finally official.  The FDA has approved our “compassionate use” IND request to give Addi and Cassi infusions of a non toxic sugar compound called 2-Hydroxypropyl-beta-Cyclodextrin or HPBCD. This Wednesday, March 18, 2009, at 7:30am and 8:30am, Addi and Cassi will have […]