Functional Loss and Mortality in Randomized Clinical Trials for Amyotrophic Lateral Sclerosis: To Combine, or Not to Combine—That is the Estimand-Reference-Cited by-同舟云学术

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Functional Loss and Mortality in Randomized Clinical Trials for Amyotrophic Lateral Sclerosis: To Combine, or Not to Combine—That is the Estimand

Published:2022-02-17 Issue:4 Volume:111 Page:817-825
ISSN:0009-9236
Container-title:Clinical Pharmacology & Therapeutics
language:en
Short-container-title:Clin Pharma and Therapeutics

Author:

van Eijk Ruben P.A.¹²,Roes Kit C.B.³,de Greef‐van der Sandt Inez⁴,van den Berg Leonard H.²,Lu Ying¹

Affiliation:

1. Department of Biomedical Data Science and Center for Innovative Study Design School of Medicine Stanford University Stanford California USA

2. Department of Neurology UMC Utrecht Brain CentreUniversity Medical Centre Utrecht Utrecht The Netherlands

3. Department of Health Evidence Section Biostatistics Radboud Medical Centre Nijmegen The Netherlands

4. Treeway BV Tilburg The Netherlands

Publisher

Wiley

Subject

Pharmacology (medical),Pharmacology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/cpt.2533

Reference40 articles.

1. Amyotrophic lateral sclerosis

2. Amyotrophic Lateral Sclerosis

3. Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved?

4. Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials

5. Prognosis for patients with amyotrophic lateral sclerosis: development and validation of a personalised prediction model

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1. Physical therapy for the management of global function, fatigue and quality of life in amyotrophic lateral sclerosis: systematic review and meta-analyses;BMJ Open;2024-08

2. Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis;Annals of Neurology;2023-06-22

3. Clinical gene therapy development for the central nervous system: Candidates and challenges for AAVs;Journal of Controlled Release;2023-05

4. Predicting risk factors for pediatric mortality in clinical trial research: A retrospective, cross-sectional study using a Healthcare Cost and Utilization Project database;Journal of Clinical and Translational Science;2023

5. Joint modeling of endpoints can be used to answer various research questions in randomized clinical trials;Journal of Clinical Epidemiology;2022-07

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