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An Eight-Month-Old Baby, First Patient Treated in Spain with Gene Therapy for Rare Metabolic Disease
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An Eight-Month-Old Baby, First Patient Treated in Spain with Gene Therapy for Rare Metabolic Disease
The Hospital Sant Joan de Déu, in Esplugues de Llobregat (Barcelona), has treated the first patient in Spain with a deficit of ornithine transcarbamilasa (OTC), a rare disease that alters the cycle of urea, with a gene therapy. The patient, 8 months old and from Lyon (France), received the therapy on December 3 and progressed favorably without side effects and with improved metabolic parameters, has explained to Europa Press the head of the Neur…
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