Cost-effectiveness analysis of gene-based therapies for patients with spinal muscular atrophy type i in australia

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    Although disease management care has improved, patients with Type I SMA treated by SOC do not achieve Model structure A Markov model was developed with five health states to model the lifespan of patients with SMA Type I, which was built in TreeAge Pro 2022 (TreeAge Software, Williamstown, MA). Information from published economic evaluations for treating patients with SMA Type I was used for building the model. The health states of "not sitting and PAV free", "sitting independently", and "walking independently" reflected the health states corresponding to SMA Type I to III. The target . . .

     

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