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

Rare Disease and Orphan Drugs Journal

Articles

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Correction: Exploratory survey study on adjunctive use of medical cannabis for Gaucher Disease (Rare Disease and Orphan Drugs Journal 2024; 10.20517/rdodj.2024.17)

Open Access Correction DOI: 10.20517/rdodj.2024.39 6 Nov 2024
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First, do no harm: the role of preclinical animal models in predicting adverse events in gene therapy clinical trials for Duchenne muscular dystrophy and X-Linked myotubular myopathy

Open Access Review DOI: 10.20517/rdodj.2024.28 3 Nov 2024
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Respiratory abnormalities in Dravet syndrome: insights from a case report and literature review

Open Access Review DOI: 10.20517/rdodj.2023.64 21 Oct 2024
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Special issue on Fabry disease - book 1: editorial

Open Access Editorial DOI: 10.20517/rdodj.2024.30 17 Oct 2024
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A landscape map of the key global rare disease organizations

Open Access Editorial DOI: 10.20517/rdodj.2024.29 16 Oct 2024
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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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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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Sustainable approaches for drug repurposing in rare diseases: recommendations from the IRDiRC Task Force

Open Access Perspective DOI: 10.20517/rdodj.2023.04 24 Apr 2023
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Submission Deadline: 30 Apr 2025

Published articles: 0

Topic: Advances in Neurofibromatosis - The Future is Bright

Submission Deadline: 28 Feb 2025

Published articles: 0

Familial Hypercholesterolemia
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

Submission Deadline: 30 Apr 2025

Published articles: 0

Topic: Advances in Neurofibromatosis - The Future is Bright

Submission Deadline: 28 Feb 2025

Published articles: 0

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