Familial NADH: Q1 oxidoreductase (complex I) deficiency: variable expression and possible treatment

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Familial NADH: Q1 oxidoreductase (complex I) deficiency: variable expression and possible treatment

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Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials.

Effectiveness of time-limited eye movement desensitization reprocessing therapy for parents of children with a rare life-limiting illness: a randomized clinical trial.

Early start of enzyme replacement therapy in pediatric male patients with classical Fabry disease is associated with attenuated disease progression.

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Familial NADH: Q1 oxidoreductase (complex I) deficiency: variable expression and possible treatment

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