For the first time, we are witnessing therapies that can fundamentally alter the course of inherited disease lifelong. The most recent breakthrough describes treating inherited disease in infants. In a case in Nature Medicine , a premature baby with a devastating genetic epilepsy syndrome achieved a 60% reduction in life-threatening seizures following treatment with an experimental therapy.

This is the first installment in a two-part series describing the opportunities for correcting inherited defects before and immediately after birth. These therapies have provided a chance for those inherited diseases to be treated. Part 1 focuses on the treatment of a newborn. In contrast, Part 2 examines novel applications in the uterus as fetuses before they are born.

Detecting Inherited Disea

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