Sage Journals: Discover world-class research

Open access

Review article

First published 2025-7

Drug Repurposing for Rare (Orphan) Diseases—Scope and Limitations

Dhavendra Kumar

Apollo Medicine

Retricted access

Review article

First published 2021-6

Challenges in Evaluating Safety and Efficacy in Drug Development for Rare Diseases: A Review for Pharmacists

Kanya K. Shah, Stephen Kogut, Angela Slitt

Journal of Pharmacy Practice

Open access

Research article

First published 2026

Knowledge Graphs and Explainable Artificial Intelligence (AI) for Drug Repurposing on Rare Diseases

Pablo Perdomo-Quinteiro, Katherine Wolstencroft, Marco Roos [...]

Neurosymbolic Artificial Intelligence

Open access

Research article

First published 2017

From Off-Label to Repurposed Drug in Non-Oncological Rare Diseases: Definition and State of the Art in Selected EU Countries

Paola Minghetti, Elena P. Lanati, Josie Godfrey [...]

The Journal of Medicine Access

Retricted access

Research article

First published 2017-3

Patient Voice in Rare Disease Drug Development and Endpoints

Linda S. Deal, Jonathan C. Goldsmith, Susan Martin [...]

Therapeutic Innovation & Regulatory Science

Open access

Review article

First published 2025

Access to drugs for rare diseases (DRD) in Canada: a comprehensive review of the provincial DRD-specific programs

Nahya Awada

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 2014-11

Opportunity Cost of Funding Drugs for Rare Diseases

Doug Coyle, Matthew C. Cheung, Gerald A. Evans

Medical Decision Making

Retricted access

Research article

First published 2019-10

The Orphan Drug Act: An Appropriate Approval Pathway for Treatments of Rare Diseases?

Michael Gabay

Hospital Pharmacy

Retricted access

Research article

First published 2025-6

Pivotal trial characteristics and types of endpoints used to support Food and Drug Administration rare disease drug approvals between 2013 and 2022

Kyungwan Hong, Bridget Nugent, Abbas Bandukwala [...]

Clinical Trials

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