23 December 2016 - The FDA has always had a tendency to announce big news late on Fridays. 

So it's perhaps not surprising that the agency issued one of the year's most important drug approvals on the evening before Christmas Eve.

The drug, Spinraza (generic name: nusinersen) treats a rare disease, spinal muscular atrophy, that is the leading cause of genetic death in infants. SMA afflicts about one in 10,000 newborns, causing degeneration of nerve cells in the spinal cord leading to weakness and paralysis. Spinraza's benefits are incontrovertible. In an analysis of 82 infants randomly assigned to either receive Spinraza or a sham needle prick, 40% of the babies achieved motor milestones including sitting, crawling, and walking. None of the babies who got the sham treatment did.

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