Dose selection for biological enzyme replacement therapy indicated for inborn errors of metabolism.

Hon, Yuen Yi; Wang, Jie; Abodakpi, Henrietta; Balakrishnan, Anand; Pacanowski, Michael; Chakder, Sushanta; Smpokou, Patroula; Donohue, Kathleen; Wang, Yow-Ming C

Hon, Yuen Yi; Wang, Jie; Abodakpi, Henrietta; Balakrishnan, Anand; Pacanowski, Michael; Chakder, Sushanta; Smpokou, Patroula; Donohue, Kathleen; Wang, Yow-Ming C.

Afiliación

Hon YY; Division of Rare Diseases and Medical Genetics, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Office of New Drugs (OND), Center of Drug Evaluation and Research (CDER), Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Wang J; Office of Clinical Pharmacology, Office of Translational Sciences, CDER, Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Abodakpi H; Office of Clinical Pharmacology, Office of Translational Sciences, CDER, Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Balakrishnan A; Office of Clinical Pharmacology, Office of Translational Sciences, CDER, Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Pacanowski M; Office of Clinical Pharmacology, Office of Translational Sciences, CDER, Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Chakder S; Division of Pharmacology and Toxicology, Office of Immunology and Inflammation, OND, CDER, Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Smpokou P; Division of Rare Diseases and Medical Genetics, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Office of New Drugs (OND), Center of Drug Evaluation and Research (CDER), Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Donohue K; Division of Rare Diseases and Medical Genetics, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Office of New Drugs (OND), Center of Drug Evaluation and Research (CDER), Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Wang YC; Office of Clinical Pharmacology, Office of Translational Sciences, CDER, Food and Drug Administration (FDA), Silver Spring, Maryland, USA.

Clin Transl Sci ; 16(12): 2438-2457, 2023 12.

Article en En | MEDLINE | ID: mdl-37735717

Asunto(s)

Terapia de Reemplazo Enzimático; Animales; Humanos; Niño; Biomarcadores

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Terapia de Reemplazo Enzimático Tipo de estudio: Prognostic_studies Límite: Animals / Child / Humans Idioma: En Revista: Clin Transl Sci Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos

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