Novel concept to evaluate efficacy of therapeutics for ALS based on patient preference

A novel composite scale (PROOF, incorporates patient’s perception of disease burden to motor functional evaluation for ALS.

Efficacy of therapeutics for neuromuscular diseases has been assessed using critical events such as survival or quantitative or semiquantitative functional scales. Most of such scales consists of items which can be clustered into several functional domains, and total scores are widely used as indices of overall function. For amyotrophic lateral sclerosis (ALS), the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) which contains 12 items belonging to four functional domains, bulbar, fine motor, gross motor and respiratory, has been widely used in clinical trials as a co-primary endpoint with survival, but it does not reflect the variability in patients’ perception of disease burden. The paper by van Eijk et al reports a new concept to evaluate the therapeutic efficacies in ALS clinical trials by considering patient preference for assessing ALSFRS-R: Patient-Ranked Order of…

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