Sage Journals: Discover world-class research

Open access

Research article

First published 2023

The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies

Katherine L. Beaverson, Daria Julkowska, Mary Catherine V. Letinturier [...]

Therapeutic Advances in Rare Disease

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

Research article

First published 2022-04-13

The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study

Debra S. Regier, Jennifer A. Weaver, Nancy Cheng [...]

Translational Science of Rare 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

Open access

Review article

First published 2026

Challenges and opportunities for the use of telehealth in rare disease diagnosis,treatment,research,and education: key opinion leader interviews by the IRDiRC telehealth task force

Melissa A. Parisi, Adam L. Hartman, Mary Catherine V. Letinturier [...]

Therapeutic Advances in Rare Disease

Open access

Research article

First published 2025

From care to cure: a patient engagement framework for rare disease and orphan drug research

Nahya Awada, Anil Varughese

Therapeutic Advances in Rare Disease

Retricted access

Research article

First published 2018-01

Research in Rare Disease: From Genomics to Proteomics

Jessica Lacoste

ASSAY and Drug Development Technologies

Open access

Review article

First published 2026-1

Privacy-by-Design with Federated Learning will drive future Rare Disease Research

Simon Süwer, Md Shihab Ullah, Niklas Probul [...]

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2016-07

The Spectrum of Caregiving in Palliative Care for Serious,Advanced,Rare Diseases: Key Issues and Research Directions

Lynn S. Adams, Jeri L. Miller, Patricia A. Grady

Journal of Palliative Medicine

Retricted access

Research article

First published 2010-03

The International LAM Registry: A Component of an Innovative Web-Based Clinician,Researcher,and Patient-Driven Rare Disease Research Platform

Michael Nurok, Ian Eslick, Carlos R. R. Carvalho [...]

Lymphatic Research and Biology

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