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Free access

Meeting report

First published 2022

Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative

Eduardo F. Tizzano, Vanessa Christie-Brown, Giovanni Baranello [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023

The Rare Diseases Clinical Research Network: a model for clinical trial readiness

Joanne M. Lumsden, Tiina K. Urv

Therapeutic Advances in Rare Disease

Retricted access

Research article

First published 2025-09

Vineland-3 Growth Scale Values: Psychometric Properties for Clinical Trial Readiness in SCN2A

Aaron J. Kaat, Lindsey Evans, Amanda N. Nili [...]

Journal of Child and Adolescent Psychopharmacology

Open access

Research article

First published 2022-5

“Ready for What?”: Timing and Speculation in Alzheimer’s Disease Drug Development

Natassia F. Brenman, Richard Milne

Science,Technology,& Human Values

Retricted access

Research article

First published 2013-4

From ClinicalTrials.gov trial registry to an analysis-ready database of clinical trial results

M Soledad Cepeda, Victor Lobanov, Jesse A Berlin

Clinical Trials

Open access

Review article

First published 2025

Enhancing and leveraging principal investigator and patient advocacy group collaboration in rare disease clinical research-meeting report from the rare Diseases Clinical Research Network

Kristen Wheeden, Sheridan Meyers, Kristin Anthony [...]

Therapeutic Advances in Rare Disease

Open access

Correction

First published 2024

Corrigendum to “The Rare Diseases Clinical Research Network: a model for clinical trial readiness”

Therapeutic Advances in Rare Disease

Retricted access

Editorial

First published 2005-5

Are We Ready for Patient-Based Effect Sizes in Clinical-Trials Research?

Harold P. Lehmann

Medical Decision Making

Retricted access

Editorial

First published 2009-11

New Dual Ventilator Modes: Are We Ready to Perform Large Clinical Trials?

Samir Jaber

Respiratory Care

Open access

Research article

First published 2022-2

Psychological Readiness to Return to Sport at 6 Months Is Higher After Bridge-Enhanced ACL Restoration Than Autograft ACL Reconstruction: Results of a Prospective Randomized Clinical Trial

Ryan M. Sanborn, Gary J. Badger, The BEAR Trial Team [...]

Orthopaedic Journal of Sports Medicine

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