Pilot trial of albuterol in spinal muscular atrophy.

The aim of this pilot study was to evaluate the effect of albuterol in children with spinal muscular atrophy (SMA). Thirteen patients (five with SMA II and eight with SMA III) were given oral albuterol for 6 months. There was a significant increase in myometry, forced vital capacity, and lean body mass between the baseline and the 6-month assessments (p < 0.05). Albuterol may have a beneficial effect in patients with SMA without causing any significant adverse effects. Larger randomized, placebo-controlled trials are needed to confirm this observation.