4.5 Article

Family, healthcare professional, and societal preferences for the treatment of infantile spinal muscular atrophy: A discrete choice experiment

Related references

Note: Only part of the references are listed.
Article Clinical Neurology

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)

Article Genetics & Heredity

Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment

Alisha Monnette et al.

Summary: The study found that patients and caregivers in SMA treatment prioritize attributes such as improvements in motor and breathing function, broad indication, oral or one-time infusion administration, and minimal risk. Some patients are willing to trade off additional gains in efficacy for a change in route of administration.

ORPHANET JOURNAL OF RARE DISEASES (2021)

Review Genetics & Heredity

Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

Tamara Dangouloff et al.

Summary: The review summarizes studies on the cost of caring for spinal muscular atrophy (SMA) patients and economic evaluations of treatments, revealing a substantial cost burden of standard care for SMA patients and high cost-effectiveness ratios of approved drugs in post-symptomatic patients at current prices. Additional prospective and independent economic studies in pre- and post-symptomatic patients are needed due to the limited number of conducted studies.

ORPHANET JOURNAL OF RARE DISEASES (2021)

Article Clinical Neurology

Parents' perspectives on nusinersen treatment for children with spinal muscular atrophy

Mette van Kruijsbergen et al.

Summary: Parents' perspectives on deciding whether their child should receive nusinersen treatment vary, from focusing on treating the disease to pursuing a good quality of life for their child. Honest and neutral communication with their physician and access to available information are key factors that help parents make decisions.

DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY (2021)

Article Medicine, General & Internal

Risdiplam in Type 1 Spinal Muscular Atrophy

Giovanni Baranello et al.

Summary: In a study involving 21 infants with type 1 spinal muscular atrophy, treatment with oral risdiplam resulted in increased levels of functional SMN protein in the blood. Infants in the high-dose group were more likely to sit without support for at least 5 seconds, and the higher dose of risdiplam was selected for the second part of the study.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

Article Clinical Neurology

Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis

Sophy T. F. Shih et al.

Summary: Newborn screening and early gene therapy significantly improves the quality and length of life for infants with spinal muscular atrophy (SMA), compared to late treatment with nusinersen. The cost-effectiveness analysis suggests that early gene therapy is dominant over late nusinersen treatment, with substantial QALY gains and cost savings per infant screened. Probability sensitivity analyses also support the value of newborn screening and gene therapy in improving outcomes for SMA infants.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2021)

Article Genetics & Heredity

The implementation of newborn screening for spinal muscular atrophy: the Australian experience

Didu S. T. Kariyawasam et al.

GENETICS IN MEDICINE (2020)

Editorial Material Clinical Neurology

Spinal muscular atrophy - the dawning of a new era

Michelle A. Farrar et al.

NATURE REVIEWS NEUROLOGY (2020)

Article Health Care Sciences & Services

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)

Editorial Material Pediatrics

Nusinersen for Spinal Muscular Atrophy Are We Paying Too Much for Too Little?

Vinay Prasad

JAMA PEDIATRICS (2018)

Article Medicine, General & Internal

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

J. R. Mendell et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Medicine, General & Internal

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

R. S. Finkel et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Clinical Neurology

A qualitative study of perceptions of meaningful change in spinal muscular atrophy

Sarah McGraw et al.

BMC NEUROLOGY (2017)

Article Economics

Choice Experiments to Quantify Preferences for Health and Healthcare: State of the Practice

Axel Muehlbacher et al.

APPLIED HEALTH ECONOMICS AND HEALTH POLICY (2016)

Article Economics

HOW DOES COST MATTER IN HEALTH-CARE DISCRETE-CHOICE EXPERIMENTS?

F. Reed Johnson et al.

HEALTH ECONOMICS (2011)

Article Statistics & Probability

Comparing Designs for Choice Experiments: A Case Study

Leonie Burgess et al.

JOURNAL OF STATISTICAL THEORY AND PRACTICE (2011)