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

Rare Disease and Orphan Drugs Journal

Articles

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Fabry nephropathy: a treatable cause of chronic kidney disease

Open Access Review DOI: 10.20517/rdodj.2023.61 10 Jul 2024
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Gene therapy for Dravet syndrome: promises and impact on disease trigger and secondary modifications

Open Access Review DOI: 10.20517/rdodj.2024.07 8 Jul 2024
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Neuropathy and pain in Fabry disease

Open Access Review DOI: 10.20517/rdodj.2024.13 7 Jul 2024
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Incorporating a new disease in the newborn screening programs in Europe: the spinal muscular atrophy case study

Open Access Opinion DOI: 10.20517/rdodj.2024.08 1 Jul 2024
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Longitudinal biomarker evaluation in Fabry disease patients receiving lentivirus-mediated gene therapy

Open Access Original Article DOI: 10.20517/rdodj.2024.14 26 Jun 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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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: 31 Dec 2024

Published articles: 0

Topic: Advancements in Diagnosis and Treatment of Familial Hypercholesterolemia

Submission Deadline: 15 Dec 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

Submission Deadline: 31 Dec 2024

Published articles: 0

Topic: Advancements in Diagnosis and Treatment of Familial Hypercholesterolemia

Submission Deadline: 15 Dec 2024

Published articles: 0

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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