Rare Disease and Orphan Drugs Journal
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Editor-in-Chief:
Daniel Scherman
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Volume 3
Volume 3, Issue 3 (2024)
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Volume 3, Issue 3 (September, 2024) – 4 articles
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Original Article
Longitudinal biomarker evaluation in Fabry disease patients receiving lentivirus-mediated gene therapy
Christiane Auray-Blais
, ... Jeffrey A. Medin
Rare Dis Orphan Drugs J
2024;3:25. DOI:
10.20517/rdodj.2024.14
Original Article | Available online: 26 Jun 2024
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Opinion
Incorporating a new disease in the newborn screening programs in Europe: the spinal muscular atrophy case study
Elena-Alexandra Tãtaru
, ... David A. Pearce
Rare Dis Orphan Drugs J
2024;3:26. DOI:
10.20517/rdodj.2024.08
Opinion | Available online: 1 Jul 2024
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Perspective
The European joint programme on rare diseases: building the rare diseases research ecosystem
Yanis Mimouni, ... Daria Julkowska
Rare Dis Orphan Drugs J
2024;3:24. DOI:
10.20517/rdodj.2024.06
Perspective | Available online: 23 Jun 2024
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Newborn screening in Mexico and Latin America: present and future
Claudia Gonzaga-Jauregui
, ... Juana Inés Navarrete-Martínez
Rare Dis Orphan Drugs J
2024;3:23. DOI:
10.20517/rdodj.2024.02
Perspective | Available online: 23 Jun 2024
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Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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Committee on Publication Ethics
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Portico
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https://www.portico.org/publishers/oae/