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Fabry nephropathy: a treatable cause of chronic kidney disease
Open Access Review 10 Jul 2024
DOI: 10.20517/rdodj.2023.61
Michael L. WestMichael L. West, ... Daniel G. Bichet
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Investigating Fabry disease - some lessons learned
Open Access Review 22 Jan 2024
DOI: 10.20517/rdodj.2023.50
Raphael SchiffmannRaphael Schiffmann
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Gene therapy for Dravet syndrome: promises and impact on disease trigger and secondary modifications
Open Access Review 8 Jul 2024
DOI: 10.20517/rdodj.2024.07
Claudia Di Berardino, ... Gaia Colasante
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The dynamic and urgent path of rare disease and orphan drug research
Open Access Editorial 15 Mar 2021
DOI: 10.20517/rdodj.2021.01
Daniel Scherman
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Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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