“Parents of Children With Rare Diseases Find Hope in For-Profit Companies” – The New York Times

san filippo Karen Aiach, and her daughter, Ornella, who has Sanfilippo syndrome, a rare disease. Ms. Aiach founded a company called Lysogene to focus on genetic therapy. Credit Ed Alcock for The New York Times

“Karen Aiach was working as a management consultant when she learned that her first daughter, Ornella, had Sanfilippo syndrome, a rare disease in which a missing enzyme causes toxic substances to build up in the body.

“Ornella was 6 months old, and the prognosis was grim: She would develop mentally and physically to between ages 2 and 4, plateau and then lose whatever she had learned. She would become extremely hyperactive and develop sleeping disorders. Most likely she would not live past 15.

“Within two years of the diagnosis, Ms. Aiach, who lives in a Paris suburb, had quit her consulting job to learn everything she could about the disease. She hired a neurobiologist to guide her in the world of medical research. And when she learned that few treatments were in the works, she founded a company called Lysogene to focus on genetic therapy.”

Read this New York Times article in its entirety here.

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