Sage Journals: Discover world-class research

Open access

Review article

First published 2024

Advancing rare disease measurement through the Rare Disease Clinical Outcome Assessment Consortium

Naomi Knoble, Lindsey T. Murray

Therapeutic Advances in Rare Disease

Retricted access

Research article

First published 2011-5

Policy Framework for Rare Disease Health Disparities

Pamela Holtzclaw Williams

Policy,Politics,& Nursing Practice

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

Free access

Review article

First published 2018-11

Pharmacovigilance of medicines for rare and ultrarare diseases

Marco Sardella, Glyn Belcher

Therapeutic Advances in Drug Safety

Retricted access

Research article

First published 2012-2

Pharmacoeconomic analyses of treatments for rare disease

Jesse C. Fishman, Grant H. Skrepnek

Pharmaceuticals Policy and Law

Free access

Review article

First published 2016-6

Creating an effective clinical registry for rare diseases

Hedwig MA D’Agnolo, Wietske Kievit, Raul J Andrade [...]

United European Gastroenterology Journal

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 2021

Utilizing telehealth to create a clinical model of care for patients with Batten disease and other rare diseases

Jessica F. Scherr, Charles Albright, Emily de los Reyes

Therapeutic Advances in Rare Disease

Open access

Review article

First published 2021

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases

Yoshitsugu Aoki, Matthew J.A. Wood

Journal of Neuromuscular Diseases

Open access

Research article

First published 2019

Role of Deleterious Rare Alleles for Acute-Onset Diffuse Interstitial Lung Disease in Collagen Diseases

Hiroshi Furukawa, Shomi Oka, Kota Shimada [...]

Therapeutic Advances in Pulmonary and Critical Care Medicine

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