Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • COMMENT

Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities

Access options

Rent or buy this article

Prices vary by article type

from$1.95

to$39.95

Prices may be subject to local taxes which are calculated during checkout

Nature Reviews Drug Discovery 20, 653-654 (2021)

doi: https://doi.org/10.1038/d41573-021-00085-9

Acknowledgements

This article is based on discussions between an expert panel convened and funded by F. Hoffmann-La Roche Ltd in November 2018.

References

  1. Luu, S. et al. Response to placebo in fragile X syndrome clinical trials: an initial analysis. Brain Sci. 10, 629 (2020).

    Article  Google Scholar 

  2. Benson, N. F. et al. W scores: background and derivation. J. Psychoed. Assess. 3, 273–277 (2018).

    Article  Google Scholar 

  3. Gargon, E., Gorst, S. L. & Williamson, P. R. Choosing important health outcomes for comparative effectiveness research: 5th annual update to a systematic review of core outcome sets for research. PLoS ONE 14, e0225980 (2019).

    Article  PubMed  Google Scholar 

  4. Sahin, M. et al. Discovering translational biomarkers in neurodevelopmental disorders. Nat. Rev. Drug Discov. 18, 235–236 (2019).

    Article  PubMed  Google Scholar 

  5. Perez-Pozuelo, I. et al. The future of sleep health: a data-driven revolution in sleep science and medicine. NPJ Digit. Med. 3, 42 (2020).

    Article  PubMed  Google Scholar 

Download references

Supplementary Information

  1. Supplementary information

Competing Interests

The authors declare no competing interests.

Nature Careers

Jobs

  • Associate or Senior Editor

    Job Title: Associate or Senior Editor, Research Cross-Journal Editorial Team, Nature Portfolio Full Time, Permanent Role Location: Shanghai, Beijin...

    New York City, New York (US)

    Springer Nature Ltd

  • 2 PhD Student Positions and 1 Post-Doctoral Position

    The DFG-funded Collaborative Research Center CRC 1644 “Phenotypic plasticity in plants - mechanisms, constraints and evolution”, hosted at the Un...

    Potsdam, Brandenburg (DE)

    Universität Potsdam

  • Fully-funded PhD positions on perovskite and tandem solar cell - ANU - world's top 30 university

    Join a world-class photovoltaics research group, contribute to the development of the next generation of solar cells at ANU- world's top 30 university

    Canberra, Australian Capital Territory (AU)

    The Australian National University- School of Engineering

  • Assistant Professor

    The Division of Immunobiology within the Department of Pathology and Immunology at Washington University School of Medicine in St. Louis is seeking...

    Saint Louis, Missouri

    Washington University in Saint Louis Division of Immunobiology

  • Become a Harvard Radcliffe Institute Fellow

    Harvard Radcliffe Institute fellows are exceptional scientists, writers, s...

    Cambridge, Massachusetts

    Harvard Radcliffe Institute

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing

Search

Quick links