Rare Disease Foundations Need To Vote 4 Hope In Pepsi Refresh

During the Vote4Hope Pepsi Refresh Rare Disease Campaign, I am making video postings on Addi and Cassi’s website to raise awareness for all rare diseases, not just the horrific disease called Niemann Pick Type C that I am fighting against on behalf of my six year old identical twins. Please take a minute to watch […]

Scientists at NIH, FDA & CDC To Hold Briefing On XMRV Retrovirus

Note: I plan to begin posting blogs about the XMRV retrovirus on Addi and Cassi’s website. Why would I start posting about XMRV when Addi and Cassi have Niemann Pick Type C disease, a fatal “childhood Alzheimer’s” like condition? People who are reading my blog for the first time need to know that all people […]

Creating Hope Act 2010 Bill Would Extend Priority Review Voucher System To Rare Pediatric Diseases

Great news for the pediatric rare disease community came out late last week — rare disease advocates please get this out on your blogs! Senators Sam Brownback (R-KS), Sherrod Brown (D-OH), and Al Franken (D-MN) are supporting the bipartisan bill S. 3697, the “Creating Hope Act of 2010.” Nancy Goodman, Executive Director of Kids v […]

IND Application Filed With FDA To Bring Cyclodextrin Into The Brain To Treat Fatal Childhood Cholesterol Disease

After months and months of work and input by doctors and researchers, Dr. Caroline Hastings at Children’s Hospital Research Center Oakland filed our second Investigational New Drug application (IND) with the FDA on July 14, 2010. The 200+ page IND filing details our request to deliver hydroxy-propel-beta-cyclodextrin (HPBCD or CYCLO) directly into Addi and Cassi’s […]

Kids Living With Dementia Diagnosis – 4 Year Old Johnathan Spenser’s Story

  Johnathan Spencer has Niemann Pick Type C disease like Addi and Cassi. Embracing every moment Thursday, April 29, 2010 By Sara Suddes (ssuddes@gilroydispatch.com):  Photo by: Lora Schraft, Staff Photographer, Gilroy Dispatch   Every morning, Johnathan Spencer’s mother dissolves the contents of a pill containing an experimental drug into a cup of water, draws the solution into a syringe […]

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