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Nature Reviews. Drug Discovery|January 9, 2020
Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges aheadErik Tambuyzer, Benjamin Vandendriessche, Christopher P Austin, et al.Nature Reviews. Drug Discovery|December 15, 2019
Therapies for rare diseases: therapeutic modalities, progress and challenges aheadErik Tambuyzer, Benjamin Vandendriessche, Christopher P Austin, et al.Acta Paediatrica (Oslo, Norway : 1992)|April 24, 2012
The need for worldwide policy and action plans for rare diseasesJohn Forman, Domenica Taruscio, Virginia A Llera, et al.Chronic Obstructive Pulmonary Diseases (Miami, Fla.)|August 30, 2017
Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare DiseasesAdam Wanner, Stephen C Groft, J Russell Teagarden, et al.Contemporary Clinical Trials|July 9, 2010
Creating a global rare disease patient registry linked to a rare diseases biorepository database: Rare Disease-HUB (RD-HUB)Yaffa R Rubinstein, Stephen C Groft, Ronald Bartek, et al.Molecular Genetics and Metabolism|June 29, 2013
Expanding research to provide an evidence base for nutritional interventions for the management of inborn errors of metabolismKathryn M Camp, Michele A Lloyd-Puryear, Lynne Yao, et al.Circulation|November 14, 2007
Inherited arrhythmias: a National Heart, Lung, and Blood Institute and Office of Rare Diseases workshop consensus report about the diagnosis, phenotyping, molecular mechanisms, and therapeutic approaches for primary cardiomyopathies of gene mutations affecting ion channel functionStephan E Lehnart, Michael J Ackerman, D Woodrow Benson, et al.JAMIA Open|January 11, 2021
The case for open science: rare diseasesYaffa R Rubinstein, Peter N Robinson, William A Gahl, et al.American Journal of Human Genetics|May 6, 2017
International Cooperation to Enable the Diagnosis of All Rare Genetic DiseasesKym M Boycott, Ana Rath, Jessica X Chong, et al.Advances in Experimental Medicine and Biology|December 8, 2017
Improved Diagnosis and Care for Rare Diseases through Implementation of Precision Public Health FrameworkGareth Baynam, Faye Bowman, Karla Lister, et al.Pageof 3