L-CARNITINE SUPPLEMENTATION IN DUCHENNE MUSCULAR DYSTROPHY STEROID-NAIVE PATIENTS: A PILOT STUDY

摘要

Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disorder characterized by progressive weakness leading to disability. Steroid administration and physiotherapy are the most useful medical interventions in ambulant patients; In DMD, muscles present a significant deficiency of L-carnitine compared to normal subjects. Nutritional supplements are used to support management. We evaluated the effect of L-carnitine supplementation in patients that were not under steroid treatment. A double blind, controlled trial was performed in 20 patients assigned to L-carnitine or placebo treatment for one year, dose of (50 mgl kg) twice daily. Evaluations were performed every 4 months and results were analyzed using ANOVA. Variables such as age, weight, and disease onset were similar in both groups. L-carnitine supplementation was well tolerated; no significant side effects were reported during and after treatment. Clinical evaluations showed no differences between groups (p>0.05). L-carnitine supplementation does not significantly improve the function of upper and lower extremities of DMD steroid-naive patients