Related references
Note: Only part of the references are listed.A Decision for Life - Treatment decisions in newly diagnosed families with spinal muscular atrophy (SMA)
Nicole Gusset et al.
EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY (2021)
Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment
Alisha Monnette et al.
ORPHANET JOURNAL OF RARE DISEASES (2021)
Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments
Tamara Dangouloff et al.
ORPHANET JOURNAL OF RARE DISEASES (2021)
Parents' perspectives on nusinersen treatment for children with spinal muscular atrophy
Mette van Kruijsbergen et al.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY (2021)
Risdiplam in Type 1 Spinal Muscular Atrophy
Giovanni Baranello et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis
Sophy T. F. Shih et al.
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2021)
The implementation of newborn screening for spinal muscular atrophy: the Australian experience
Didu S. T. Kariyawasam et al.
GENETICS IN MEDICINE (2020)
Spinal muscular atrophy - the dawning of a new era
Michelle A. Farrar et al.
NATURE REVIEWS NEUROLOGY (2020)
The Whole Game is Changing and You've Got Hope: Australian Perspectives on Treatment Decision Making in Spinal Muscular Atrophy
Michelle A. Farrar et al.
PATIENT-PATIENT CENTERED OUTCOMES RESEARCH (2020)
Evaluating Benefit-risk Decision-making in Spinal Muscular Atrophy: A First-ever Study to Assess Risk Tolerance in the SMA Patient Community
Rosangel Cruz et al.
CLINICAL THERAPEUTICS (2019)
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
Darryl C. De Vivo et al.
NEUROMUSCULAR DISORDERS (2019)
Nusinersen for Spinal Muscular Atrophy Are We Paying Too Much for Too Little?
Vinay Prasad
JAMA PEDIATRICS (2018)
Ethical Challenges Confronted When Providing Nusinersen Treatment for Spinal Muscular Atrophy
Alyssa M. Burgart et al.
JAMA PEDIATRICS (2018)
Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients
Francoise Rouault et al.
NEUROMUSCULAR DISORDERS (2017)
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
J. R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
R. S. Finkel et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
A qualitative study of perceptions of meaningful change in spinal muscular atrophy
Sarah McGraw et al.
BMC NEUROLOGY (2017)
Statistical Methods for the Analysis of Discrete Choice Experiments: A Report of the ISPOR Conjoint Analysis Good Research Practices Task Force
A. Brett Hauber et al.
VALUE IN HEALTH (2016)
Choice Experiments to Quantify Preferences for Health and Healthcare: State of the Practice
Axel Muehlbacher et al.
APPLIED HEALTH ECONOMICS AND HEALTH POLICY (2016)
Constructing Experimental Designs for Discrete-Choice Experiments: Report of the ISPOR Conjoint Analysis Experimental Design Good Research Practices Task Force
F. Reed Johnson et al.
VALUE IN HEALTH (2013)
HOW DOES COST MATTER IN HEALTH-CARE DISCRETE-CHOICE EXPERIMENTS?
F. Reed Johnson et al.
HEALTH ECONOMICS (2011)
Conjoint Analysis Applications in Health-a Checklist: A Report of the ISPOR Good Research Practices for Conjoint Analysis Task Force
John F. P. Bridges et al.
VALUE IN HEALTH (2011)
Comparing Designs for Choice Experiments: A Case Study
Leonie Burgess et al.
JOURNAL OF STATISTICAL THEORY AND PRACTICE (2011)