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FDA OKs first treatment for rare genetic disorder

Posted at 10:03 PM, Dec 23, 2016

WASHINGTON (AP) — The Food and Drug Administration has approved the first treatment for children and adults with spinal muscular atrophy, a rare genetic disorder marked by progressive muscle weakness that’s the most common genetic cause of death in infants.

The agency said Friday that it approved the drug, Spinraza, after granting it fast-track status. It was developed by Ionis Pharmaceuticals Inc. of Carlsbad, California, and Biogen Inc. of Cambridge, Massachusetts. Biogen will market it.

The organization Cure SMA says spinal muscular atrophy affects about 1 in 10,000 babies, and about 1 in every 50 Americans is a genetic carrier. The FDA noted the disorder, which affects the motor nerve cells in the spinal cord, can impact people of any age.