Editor-in-Chief: Daniel Scherman
Submission to first decision: 72 days

Rare Disease and Orphan Drugs Journal

Articles

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Long-term treatment with insulin-like growth factor-1 in Phelan-McDermid syndrome: a case report

Open Access Case Report DOI: 10.20517/rdodj.2023.53 5 May 2024
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Biomarkers in anderson-Fabry disease: what should we use in the clinical practice?

Open Access Review DOI: 10.20517/rdodj.2023.56 24 Apr 2024
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Analysis of genomics implementation in newborn screening for inherited metabolic disorders: an IRDiRC initiative

Open Access Opinion DOI: 10.20517/rdodj.2023.52 23 Apr 2024
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The inflammatory pathogenetic pathways of Fabry nephropathy

Open Access Review DOI: 10.20517/rdodj.2023.37 17 Apr 2024
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Gastrointestinal involvement in Fabry disease

Open Access Review DOI: 10.20517/rdodj.2023.46 28 Mar 2024
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Connecting academia and industry for innovative drug repurposing in rare diseases: it is worth a try

Open Access Perspective DOI: 10.20517/rdodj.2023.06 9 Apr 2023
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The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis

Open Access Review DOI: 10.20517/rdodj.2022.04 7 Apr 2022
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Enzyme replacement therapy: current challenges and drug delivery prospects via extracellular vesicles

Open Access Editorial DOI: 10.20517/rdodj.2022.09 3 Jul 2022
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Topic: Prader-Willi Syndrome: Clinical, Genetic, and Endocrine Research

Submission Deadline: 15 Nov 2024

Published articles: 0

Topic: Sickle Cell Disease(SCD): Progress in Research and Insights

Submission Deadline: 31 Aug 2024

Published articles: 0

We consistently welcome suggestions for improvements from both readers and authors (send an e-mail to preprint@scierxiv.com).
During the interview, Dr. Hagerman elaborated on the key points of her article, current research, and prospects within the field of Fragile X Syndrome, and offered suggestions for the future development of the journal.
During the interview, Dr. Politei talked about the highlight of this article, his current research, prospects in the field of Fabry diseases, suggestions for the future development of the journal.
Rare Disease and Orphan Drugs Journal is delighted to announce support for the 2024 Global Rare Disease Research Symposium

Topic: Prader-Willi Syndrome: Clinical, Genetic, and Endocrine Research

Submission Deadline: 15 Nov 2024

Published articles: 0

Topic: Sickle Cell Disease(SCD): Progress in Research and Insights

Submission Deadline: 31 Aug 2024

Published articles: 0

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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https://www.portico.org/publishers/oae/