Thank You Johnson & Johnson For Helping Sick Kids

I am happy to report to everyone that Johnson & Johnson really does care about children, especially Addi and Cassi and kids suffering from Niemann Pick Type C disease. After I posted my previous blog and contacted Johnson & Johnson’s PR department about our situation with the FDA and obtaining cyclodextrin safety data from their […]

Dear Johnson & Johnson, do kids really matter to you?

After weeks of going back and forth with the U.S. Food and Drug Administration (FDA) on our request to give Addi and Cassi hydroxypropyl-ß-cyclodextrin (HP-ß-CD) or "CYCLO" infusions, we finally have a Type A meeting scheduled on February 26, 2009, from Noon-1pm to discuss the clinical hold the FDA put on our "compassionate use" treatment […]

World Rare Disease Day 2009 Genes Video

I made this video for World Rare Disease Day 2008 to bring global attention to the fact that Rare Disease is not so rare — it can happen to anyone or any family. Although each Rare Disease affects a small number of people, taken all together rare diseases affect millions of people in the world. […]

FDA Reviewing Investigational New Drug Application For 5 Year Old Twins Stricken With Fatal Cholesterol Disorder

I have always believed there would be a drug sitting on shelf somewhere that could help save the lives of our 5 year old twin daughters, Addi and Cassi. What I never imagined was a sugar compound used in the food processing industry to extract cholesterol and make cholesterol free products like salad dressing could […]

Indian Boy To Recieve Mesenchymal Intracranial Stem Cell Transplant

There is a brave little boy in India who is going to make history this week. His name is Aaditya (ah-dah-tee-ah) and right now this little boy is at BGS Global Hospital in Bangalore, India, being prepped for one of the first intracranial stem cell transplants ever conducted on children. The procedure was approved by the Indian government a […]

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