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Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD).

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第一作者： Louis S,Matza

第一单位： Patient-Centered Research, Evidera, 7101 Wisconsin Avenue, Suite 1400, Bethesda, MD, 20814, USA. louis.matza@evidera.com.

作者： Louis S,Matza ^[1] ; Katie D,Stewart ^[2] ; Marie,Fournier ^[3] ; Donna,Rowen ^[4] ; Robin,Lachmann ^[5] ; Maurizio,Scarpa ^[6] ; Eugen,Mengel ^[7] ; Travis,Obermeyer ^[8] ; Evren,Ayik ^[9] ; Fernando,Laredo ^[10] ; Ruth,Pulikottil-Jacob ^[11]

作者单位： Patient-Centered Research, Evidera, 7101 Wisconsin Avenue, Suite 1400, Bethesda, MD, 20814, USA. louis.matza@evidera.com. ^[1] Patient-Centered Research, Evidera, 7101 Wisconsin Avenue, Suite 1400, Bethesda, MD, 20814, USA. ^[2] Sanofi, Chilly-Mazarin, France. ^[3] School of Health and Related Research, University of Sheffield, Sheffield, UK. ^[4] University College London Hospitals, London, UK. ^[5] Centro Coordinamento Regionale Malattie Rare, Azienda Sanitaria Universitaria del Friuli Centrale, Udine, Italy. ^[6] SphinCS-Institute of Clinical Science for Lysosomal Storage Diseases, Hochheim, Germany. ^[7] NNPDF, Charlotte, NC, USA. ^[8] , Fresno, CA, USA. ^[9] Sanofi, São Paulo, Brazil. ^[10] Sanofi, Thames Valley Park, Reading, UK. ^[11]

关键词 Acid sphingomyelinase deficiency Pediatric utility Rare disease TTO Time trade-off Utility