Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency.

Diaz, George A; Schulze, Andreas; McNutt, Markey C; Leão-Teles, Elisa; Merritt, J Lawrence; Enns, Gregory M; Batzios, Spyros; Bannick, Allison; Zori, Roberto T; Sloan, Leslie S; Potts, Susan L; Bubb, Gillian; Quinn, Anthony G

Diaz, George A; Schulze, Andreas; McNutt, Markey C; Leão-Teles, Elisa; Merritt, J Lawrence; Enns, Gregory M; Batzios, Spyros; Bannick, Allison; Zori, Roberto T; Sloan, Leslie S; Potts, Susan L; Bubb, Gillian; Quinn, Anthony G.

Afiliación

Diaz GA; Icahn School of Medicine at Mount Sinai, New York City, New York, USA.
Schulze A; University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada.
McNutt MC; UT Southwestern Medical Center, Dallas, Texas, USA.
Leão-Teles E; Centro Hospitalar de São João, Porto, Portugal.
Merritt JL; University of Washington, Seattle, Washington, USA.
Enns GM; Stanford University, Stanford, California, USA.
Batzios S; Great Ormond Street Hospital NHS Trust, London, UK.
Bannick A; Children's Hospital of Michigan, Wayne State University, Detroit, Michigan, USA.
Zori RT; University of Florida, Gainesville, Florida, USA.
Sloan LS; Aeglea BioTherapeutics, Inc., Austin, Texas, USA.
Potts SL; Aeglea BioTherapeutics, Inc., Austin, Texas, USA.
Bubb G; Aeglea BioTherapeutics, Inc., Austin, Texas, USA.
Quinn AG; Aeglea BioTherapeutics, Inc., Austin, Texas, USA.

J Inherit Metab Dis ; 44(4): 847-856, 2021 07.

Article en En | MEDLINE | ID: mdl-33325055

Asunto(s)

Arginasa/genética; Arginasa/uso terapéutico; Arginina/sangre; Hiperargininemia/tratamiento farmacológico; Adolescente; Adulto; Arginasa/efectos adversos; Arginasa/sangre; Arginina/metabolismo; Niño; Preescolar; Manejo de la Enfermedad; Femenino; Humanos; Hiperamonemia/etiología; Hiperargininemia/sangre; Hiperargininemia/genética; Hiperargininemia/metabolismo; Masculino; Proteínas Recombinantes/efectos adversos; Proteínas Recombinantes/uso terapéutico; Estados Unidos; Vómitos/etiología; Adulto Joven

Palabras clave

ARG1-D; arginase 1 deficiency; human enzyme; hyperammonemia; hyperargininemia; pegzilarginase; spasticity

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Arginasa / Arginina / Hiperargininemia Tipo de estudio: Clinical_trials / Etiology_studies Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male País/Región como asunto: America do norte Idioma: En Revista: J Inherit Metab Dis Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos

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