Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study.

Dimachkie, Mazen M; Kishnani, Priya S; Ivanescu, Cristina; Flore, Giulio; Gwaltney, Chad; van der Beek, Nadine A M E; Hamed, Alaa; An Haack, Kristina; Pollissard, Laurence; Baranowski, Eileen; Sparks, Susan E; DasMahapatra, Pronabesh

Dimachkie, Mazen M; Kishnani, Priya S; Ivanescu, Cristina; Flore, Giulio; Gwaltney, Chad; van der Beek, Nadine A M E; Hamed, Alaa; An Haack, Kristina; Pollissard, Laurence; Baranowski, Eileen; Sparks, Susan E; DasMahapatra, Pronabesh.

Afiliação

Dimachkie MM; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
Kishnani PS; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
Ivanescu C; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
Flore G; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
Gwaltney C; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
van der Beek NAME; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
Hamed A; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
An Haack K; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
Pollissard L; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
Baranowski E; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
Sparks SE; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen
DasMahapatra P; University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Departmen

Neurol Clin Pract ; 13(5): e200181, 2023 Oct.

Article em En | MEDLINE | ID: mdl-37559825

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Clinical_trials / Diagnostic_studies / Prognostic_studies Idioma: En Revista: Neurol Clin Pract Ano de publicação: 2023 Tipo de documento: Article

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