A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease.

Harmatz, Paul; Whitley, Chester B; Wang, Raymond Y; Bauer, Mislen; Song, Wenjie; Haller, Christine; Kakkis, Emil

Harmatz, Paul; Whitley, Chester B; Wang, Raymond Y; Bauer, Mislen; Song, Wenjie; Haller, Christine; Kakkis, Emil.

Afiliação

Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, United States. Electronic address: Pharmatz@mail.cho.org.
Whitley CB; University of Minnesota, Minneapolis, MN, United States.
Wang RY; Children's Hospital of Orange County, Orange, CA, United States.
Bauer M; Miami Children's Hospital, Miami, FL, United States.
Song W; Ultragenyx Pharmaceutical Inc., Novato, CA, United States.
Haller C; Ultragenyx Pharmaceutical Inc., Novato, CA, United States.
Kakkis E; Ultragenyx Pharmaceutical Inc., Novato, CA, United States.

Mol Genet Metab ; 123(4): 488-494, 2018 04.

Article em En | MEDLINE | ID: mdl-29478819

Assuntos

Glucuronidase/administração & dosagem; Mucopolissacaridose VII/terapia; Proteínas Recombinantes/administração & dosagem; Adolescente; Adulto; Criança; Feminino; Seguimentos; Glucuronidase/deficiência; Humanos; Masculino; Mucopolissacaridose VII/enzimologia; Mucopolissacaridose VII/patologia; Prognóstico; Adulto Jovem

Palavras-chave

Blind Start study design; Enzyme replacement therapy; MPS VII; Multi-domain responder index; Urinary GAG; Vestronidase alfa

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Proteínas Recombinantes / Mucopolissacaridose VII / Glucuronidase Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2018 Tipo de documento: Article

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