The question of determining the severity of autoimmune myasthenia has become central since the arrival of innovative therapies on the market. Methodologists working with the pharmaceutical company UCB have focused on the assessment parameters that are most relevant to the patients themselves (patient-related outcome measures, or PROMs):
These PROMs were compared with conventional functional scores and put into perspective with indices of muscle strength and fatigue,
This study of 43 myasthenic patients was part of a phase IIa clinical trial testing rozanolizumab,
- A post-hoc analysis of the data demonstrated the relevance and, above all, the complementarity of these PROMs compared with conventional tools for assessing myasthenia.
- Dystrophies musculaires congénitales. (DMC)
- Chimeric proteins improve peripheral neuropathy in the mouse model of CMD 1A
