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Effet du traitement par la leptine recombinante chez les enfants atteints de diabète lipoatrophique
Effect of recombinant human leptin (r-metHuLeptin) in children with congenital lipodystrophy
A phase 1/11 randomised, controlled study of a treatment for patients with Neuronopathic Gaucher disease
Ensayo clínico de terapia de reemplazamiento enzimático con replagal en niños menores de 18 años con enfermedad de Fabry
A clinical trial of replagal enzyme replacement therapy in children aged <18 years with fabry disease.
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