Switching to Glycerol Phenylbutyrate in 48 Patients with Urea Cycle Disorders: Clinical Experience in Spain.
Elena Martín-HernándezPilar Quijada-FrailePatricia CorrecherSilvia MeavillaPaula Sánchez-PintosJavier de Las Heras MonteroJavier Blasco-AlonsoLucy DoughertyAna MarquezLuis Peña-QuintanaElvira Cañedo VillarroyaMaría Concepción García-JimenezPedro Juan Moreno LozanoMercedes Murray HurtadoMaría Camprodon GómezDelia Barrio-CarrerasMariela de Los SantosMireia Del ToroMaria-Luz CouceIsidro VitoriaMontserrat Morales ConejoMarcello BellusciPublished in: Journal of clinical medicine (2022)
This study demonstrates the safety of direct switching from other nitrogen scavengers to GPB in clinical practice, which improves efficacy, metabolic control, and RAE compared to previous treatments.
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