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Preparing for the age of therapeutic trials in frontotemporal lobar degeneration
  1. David Irwin
  1. Penn Frontotemporal Degeneration Center, Department of Neurology, University of Pennsylvania, Philadelphia, PA, USA
  1. Correspondence to Dr David Irwin, Neurology, University of Pennsylvania, Philadelphia, Pennsylvania, USA; dirwin{at}pennmedicine.upenn.edu

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There is a rapidly growing field of therapeutic development for familial forms of FTLD and Peakman et al address the important need for adequate clinical tools to measure treatment effect.

Frontotemporal lobar degeneration (FTLD) comprises a spectrum of heterogenous clinicopathological neurodegenerative disorders and neuropathological examination at autopsy remains the gold standard for diagnosis.1 However, detection of pathogenic mutations in genes known to associate with either FTLD-Tau or FTLD-TDP neuropathology in ~20%–30% of all FTLD with familial disease provides an accurate antemortem molecular diagnosis in these individuals, even in the presymptomatic stage. Therefore, several clinical trials for agents targeting disease-specific mechanisms associated with forms of familial FTLD are planned or currently underway. Reliable clinical outcome measures to detect and track early disease are crucial to facilitate the success of these efforts. Peakman et al2 perform a detailed cross-sectional and longitudinal analysis of two commonly used clinical rating scales (ie, extended clinical dementia rating scale (CDR+NACC FTLD) and the Frontotemporal Dementia Rating Scale (FRS)) in the large international …

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Footnotes

  • Contributors I am the sole author of the manuscript.

  • Funding This work was supported by grants from NIH grants R01-NS109260, P01-AG066597, P30-AG10124, U19-AG062418 and the Penn Institute on Aging.

  • Disclaimer DJI receives research support to conduct therapeutic trials from Alector and Prevail.

  • Competing interests None declared.

  • Provenance and peer review Commissioned; internally peer reviewed.

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