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Novel concept to evaluate efficacy of therapeutics for ALS based on patient preference
  1. Atsushi Hashizume1,2,
  2. Masahisa Katsuno1,2
  1. 1Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya, Japan
  2. 2Department of Clinical Research Education, Nagoya University Graduate School of Medicine, Nagoya, Japan
  1. Correspondence to Dr Masahisa Katsuno, Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya, Japan; ka2no{at}

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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 …

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  • Contributors AH contributed to drafting/revising the manuscript. MK contributed to revising the manuscript.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests None declared.

  • Provenance and peer review Commissioned; internally peer reviewed.

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