Merlini 2007.
Methods | Randomised, placebo‐controlled, double‐blind trial |
Participants | 110 participants with SMA types II or III, aged ≥ 4 years |
Interventions | Acetyl‐L‐carnitine 50 mg/kg/day (maximum 3 g/day) (route not mentioned) or placebo Duration of treatment: 9 months Follow‐up: 12 months |
Outcomes | Change in muscle strength arm and leg (myometry), change in functional score (time to walk 10 m and to arise from floor), change in pulmonary function (FVC), change in quality of life (Short Form‐36 Health Survey questionnaire (SF‐36) or Childhood Health Assessment Questionnaire. |
Notes | Study status unknown (last report on trial notified in 2007). No results available |