Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities-Reference-Cited by-同舟云学术

Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities

Published:2021-05-17 Issue:9 Volume:20 Page:653-654
ISSN:1474-1776
Container-title:Nature Reviews Drug Discovery
language:en
Short-container-title:Nat Rev Drug Discov

Author:

Krishnan Michelle L.,Berry-Kravis Elizabeth,Capal Jamie K.,Carpenter Randall,Gringras Paul,Hipp Joerg F.,Miller Meghan T.,Mingorance Ana,Philpot Benjamin D.,Pletcher Mathew T.,Rotenberg Alexander,Tjeertes Jorrit,Wang Paul P.,Willgoss Tom,de Wit Marie-Claire,Jeste Shafali S.

Publisher

Springer Science and Business Media LLC

Subject

Drug Discovery,Pharmacology,General Medicine

Link

https://www.nature.com/articles/d41573-021-00085-9.pdf

Reference5 articles.

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

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

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).

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

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).

Cited by 8 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Parent‐reported outcome measures evaluating communication in individuals with rare neurodevelopmental disorders: A systematic review;International Journal of Language & Communication Disorders;2024-08-14

2. Leveraging the power of community to strengthen clinical trials for rare genetic syndromes;The Transmitter;2024

3. A Prospective, Longitudinal Study of Caregiver-Reported Adaptive Skills and Function of Individuals with HNRNPH2-related Neurodevelopmental Disorder;Advances in Neurodevelopmental Disorders;2023-08-07

4. Developing Angelman syndrome-specific clinician-reported and caregiver-reported measures to support holistic, patient-centered drug development;Orphanet Journal of Rare Diseases;2023-06-22

5. Disruption of Intracellular Calcium Homeostasis Leads to ERLIN2-Linked Hereditary Spastic Paraplegia in Patient-Derived Stem Cell Models;Human Mutation;2023-06-16