They had promising gene therapies for their daughter's ultra-rare disease. But how to tell if one worked?
About two years ago, Majid Jafar, CEO of the 50-year-old Emirati oil firm Crescent Petroleum, found himself with a problem of his own making, one that he was happy to have.
In 2014, Jafar and his wife, Lynn Barghout Jafar, had a daughter, Alia. She came home more than 8 pounds and healthy. But on day 19, Alia suffered 10 seizures. The Jafars rushed her to doctors, who several months later finally diagnosed her with CDKL5 deficiency disorder, or CDD, a disease that just a decade prior researchers didn’t know existed.
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