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 pediatric neurologist who was involved in the clinical trials for the drug for spinal muscular atrophy.
