Consensus study establishes a standardized outcome measures toolkit in adult spinal muscular atrophy, suggesting improved clinical monitoring and higher real-world evidence quality.
The implementation of the consensus-derived OM toolkit will improve monitoring and assessment of adult SMA patients, and enrich the quality of real-world evidence. Regular updates to the toolkit must be considered as new evidence becomes available.
No takes yet. Share an insight, caveat, or question.
Slayter et al. (2021) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: