相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Health-related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study
Erik Landfeldt et al.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY (2016)
AdViSHE: A Validation-Assessment Tool of Health-Economic Models for Decision Makers and Model Users
P. Vemer et al.
PHARMACOECONOMICS (2016)
Quantifying the burden of caregiving in Duchenne muscular dystrophy
Erik Landfeldt et al.
JOURNAL OF NEUROLOGY (2016)
Development and psychometric analysis of the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT)
Erik Landfeldt et al.
NEUROMUSCULAR DISORDERS (2015)
Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries
Todd Gammie et al.
PLOS ONE (2015)
The burden of Duchenne muscular dystrophy An international, cross-sectional study
Erik Landfeldt et al.
NEUROLOGY (2014)
Shining a light in the black box of orphan drug pricing
Eline Picavet et al.
ORPHANET JOURNAL OF RARE DISEASES (2014)
Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy
Valeria Ricotti et al.
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2013)
Cost-Effectiveness Assessment of Orphan Drugs A Scientific and Political Conundrum
Steven Simoens et al.
APPLIED HEALTH ECONOMICS AND HEALTH POLICY (2013)
Evidence-Based Path to Newborn Screening for Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2012)
Motor and respiratory heterogeneity in Duchenne patients: Implication for clinical trials
Veronique Humbertclaude et al.
EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY (2012)
Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?
Wills Hughes-Wilson et al.
ORPHANET JOURNAL OF RARE DISEASES (2012)
Modeling Good Research Practices-Overview: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-1
J. Jaime Caro et al.
VALUE IN HEALTH (2012)
Regression models for analyzing costs and their determinants in health care: an introductory review
Dario Gregori et al.
INTERNATIONAL JOURNAL FOR QUALITY IN HEALTH CARE (2011)
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management
Katharine Bushby et al.
LANCET NEUROLOGY (2010)
Assessing the economic challenges posed by orphan drugs
Michael F. Drummond et al.
INTERNATIONAL JOURNAL OF TECHNOLOGY ASSESSMENT IN HEALTH CARE (2007)
Managing Duchenne muscular dystrophy - The additive effect of spinal surgery and home nocturnal ventilation in improving survival
Michelle Eagle et al.
NEUROMUSCULAR DISORDERS (2007)
Multiattribute and single-attribute utility functions for the health utilities index mark 3 system
D Feeny et al.
MEDICAL CARE (2002)