2 March 2017 - Two days before Christmas, the Food and Drug Administration gave Thomas Crawford an unexpected gift: approval of the first treatment ever for a devastating genetic disease that causes muscle wasting in babies and often results in death at an early age.

The drug “is nothing short of oh-my-God amazing” when given to infants who have not yet had symptoms, said Crawford, a Johns Hopkins paediatric neurologist who was involved in the clinical trials for the drug for spinal muscular atrophy.

What really surprised Crawford was the timing. The FDA approved Spinraza, as the treatment is called, in three months, five months ahead of its deadline. “That was very, very fast,” he said.

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