FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease

Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. While we have not received the official letter by mail (probably next week), it appears our Orphan […]

FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community

Actelion, the makers of Zavesca, today put out a brief statement saying that the U.S. Food and Drug Administration did not approve Zavesca and wants more information from the company on treating Niemann Pick Type C, a rare and dementing neurological disease that afflicts our six year old identical twins, Addi and Cassi. Niemann Pick Type C disease is caused […]

FDA Orphan Drug Workshop Makes Filing Applications Easy – Even For Rare Disease Patient Advocates!

At a historic orphan drug workshop held by the U.S. Food and Drug Administration Feb. 25-26 and before World Rare Disease Day 2010, we filed a momentous orphan drug application with the FDA for the treatment of Niemann Pick Type C disease with cyclodextrin. Our filing occurred in Claremont, CA, at the Keck Graduate Institute’s Center […]

NIH Announces Therapeutic Trial With Supplement N-Acetyl Cysteine (NAC) To Treat Fatal Children’s Cholesterol Disease

You’ve got to LOVE supplements especially one called N-Acetyl Cysteine (NAC) when it can possibly help treat children afflicted with a rare and fatal cholesterol disease called Niemann Pick Type C (NPC). Considering there has only been only one clinical trial for Niemann Pick Type C ever conducted at the NIH (for a drug called […]

Hope, Faith and Love – Our Nations Robotic Heros!

We have returned from the National Chemical Genomics Center (NCGC) and are busy working on pushing forward new therapy ideas for Addi and Cassi and Niemann Pick Type C disease using state-of-the-art cheminformatics methods. The robotic equipment the NCGC has established for testing cells and profiling compounds for large collections of chemicals is incredible.  The […]

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