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document
Rett syndrome : randomized controlled trial of L-carnitine . Rett syndrome is a severe neurodevelopmental disorder of unknown etiology , occurring almost exclusively in female patients . The etiology and functional significance of plasma carnitine deficiency seen in some patients with Rett syndrome is unknown . To investigate whether L-carnitine might be of benefit in Rett syndrome , a randomized , placebo-controlled , double-blind crossover trial of L-carnitine has been completed in 35 subjects . Eight-week treatment phases were completed for both a placebo and L-carnitine . Outcome was measured by parents/caregivers and at medical follow-up using three established tools : the Rett Syndrome Motor Behavioral Assessment , the Hand Apraxia Scale , and the Patient Well-Being Index . Analysis comparing change between baseline and week 8 of treatment for L-carnitine and the placebo showed that both parents/caregivers and medical follow-up detected improvements in the subjects ' well-being . In addition , medical review showed an improvement on the Hand Apraxia Scale for a higher proportion of girls on L-carnitine . Identification of predictors of clinical improvement has been limited by the power of the study . These findings suggest that L-carnitine is of benefit in some patients with Rett syndrome . While L-carnitine did not lead to major functional changes in ability , the type of changes reported could still have a substantial impact on the girls and their families . Information is still needed , however , to determine if only subgroups of girls with the disorder are responsive to L-carnitine and the appropriate duration of therapy .
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Intervention_Pharmacological
L-carnitine
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Intervention_Pharmacological
L-carnitine
515
Intervention_Control
placebo-controlled
516
Intervention_Pharmacological
L-carnitine
517
Intervention_Control
placebo
518
Intervention_Pharmacological
L-carnitine
519
Intervention_Pharmacological
L-carnitine
520
Intervention_Control
placebo
521
Intervention_Pharmacological
L-carnitine
522
Intervention_Pharmacological
L-carnitine
523
Intervention_Pharmacological
L-carnitine
524
Outcome_Mental
the Rett Syndrome Motor Behavioral Assessment
525
Outcome_Mental
the Hand Apraxia Scale
526
Outcome_Other
the
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Outcome_Mental
Patient Well-Being Index
528
Outcome_Mental
well-being
529
Outcome_Mental
Hand Apraxia Scale
530
Participant_Condition
Rett syndrome :
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Participant_Condition
female patients .