Open Access<p>Estimation Of The Quality Of Life Benefits Associated With Treatment For Spinal Muscular Atrophy</p>
Author(s) -
Andrew Lloyd,
Robin Thompson,
Katy Gallop,
Megan Teynor
Publication year - 2019
Publication title -
clinicoeconomics and outcomes research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.591
H-Index - 27
ISSN - 1178-6981
DOI - 10.2147/ceor.s214084
Subject(s) - sma* , spinal muscular atrophy , medicine , quality of life (healthcare) , neuromuscular disease , pediatrics , disease , atrophy , weakness , physical medicine and rehabilitation , physical therapy , surgery , mathematics , nursing , combinatorics
Spinal muscular atrophy (SMA) is a rare, genetic, progressive neuromuscular disorder characterized by severe muscle atrophy and weakness and is a leading genetic cause of death in infants and children. Nusinersen was the first treatment targeting the underlying cause of disease approved by the FDA, EMA and other countries for patients with SMA. There are currently very limited data available on the health-related quality of life (HRQoL) burden of SMA suitable for use in a cost-effectiveness analysis.