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Good news for rare disease?

The mother of young twins with a rare genetic disease is seeking approval from the U.S. Food and Drug Administration to administer linkurl:a non-prescription compound;http://www.the-scientist.com/blog/display/55377/ directly into the brains of her girls based on recent findings showing the compound dramatically improves cats with the disease. It may seem unusual for a parent to fill out such an application to the FDA, but Chris Hempel, who has two 6-year old children suffering from Niemann-Pick

Alison McCook
The mother of young twins with a rare genetic disease is seeking approval from the U.S. Food and Drug Administration to administer linkurl:a non-prescription compound;http://www.the-scientist.com/blog/display/55377/ directly into the brains of her girls based on recent findings showing the compound dramatically improves cats with the disease. It may seem unusual for a parent to fill out such an application to the FDA, but Chris Hempel, who has two 6-year old children suffering from Niemann-Pick Type C (NPC), has practice. Last month, the FDA approved her orphan drug designation application for the compound in question, cyclodextrin, widely employed by the food and chemical industries, and used as a drug solubilizer. "I'm so excited," Hempel told The Scientist. "It's been a long process."
Addi and Cassi getting their intravenous infusions
Image: Chris Hempel
Hempel has been giving her twin girls regular intravenous infusions of cyclodextrin to stop or stall the progression of...
The Scientist



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