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Articles (465)

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1-10 of 465 results for rare disease clinical trials

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Select article: Clinical Trials in Rare Disease

Retricted access

Research article

First published 2013-9

Clinical Trials in Rare Disease

Erika F. Augustine, Heather R. Adams, Jonathan W. Mink

Journal of Child Neurology

Select article: Tackling rare diseases: Clinical trials on chips

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Review article

First published 2020-7

Tackling rare diseases: Clinical trials on chips

Sandra H Blumenrath, Bo Y Lee, Lucie Low [...]

Experimental Biology and Medicine

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

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

Select article: Designing Clinical Trials to Study Rare Disease Treatment

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Other

First published 1998-1

Designing Clinical Trials to Study Rare Disease Treatment

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

Select article: Bayesian sequential decision-making for rare disease clinical trials

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Research article

First published 2025-9

Bayesian sequential decision-making for rare disease clinical trials

Yuan Gao, Jianling Bai, Feng Chen

Technology and Health Care

Select article: Clinical Trials for Rare Lung Diseases: Lessons from Lymphangioleiomyomatosis

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Research article

First published 2010-03

Clinical Trials for Rare Lung Diseases: Lessons from Lymphangioleiomyomatosis

Brent Kinder, Francis X. McCormack

Lymphatic Research and Biology

Select article: N-of-1 Clinical Trials: A Scientific Approach to Personalized Medicine for Patients with Rare Retinal Diseases Such as Retinitis Pigmentosa

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Review article

First published 2021-11

N-of-1 Clinical Trials: A Scientific Approach to Personalized Medicine for Patients with Rare Retinal Diseases Such as Retinitis Pigmentosa

Marco A. Zarbin, Gary Novack

Journal of Ocular Pharmacology and Therapeutics

Select article: A novel,hybrid,single- and multi-site clinical trial design for CLN3 disease,an ultra-rare lysosomal storage disorder

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Research article

First published 2019-10

A novel,hybrid,single- and multi-site clinical trial design for CLN3 disease,an ultra-rare lysosomal storage disorder

Heather R Adams, Sara Defendorf, Amy Vierhile [...]

Clinical Trials

Select article: Orphan Drug Development — International Program and Study Design Issues

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Other

First published 1998-1

Orphan Drug Development — International Program and Study Design Issues

Marlene E. Haffner

Therapeutic Innovation & Regulatory Science

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

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

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