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. 2021 Feb 12;19(4):501–520. doi: 10.1007/s40258-020-00624-2
No study has systematically reviewed the literature for estimates of costs of illness of spinal muscular atrophy.
The body of literature of costs of illness of spinal muscular atrophy is limited to a few geographical settings and characterized by considerable variability across disease phenotypes.
Our review helps inform economic evaluations and future cost research, and highlights the substantial unmet medical need in spinal muscular atrophy.