Rare Disease and Orphan Drugs Journal
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Editor-in-Chief: Daniel Scherman
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Rare Disease and Orphan Drugs Journal
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Editor-in-Chief: Daniel Scherman
Submission to first decision: 72 days
Home Articles Special Issues Volumes Webinars Videos

Rare Disease and Orphan Drugs Journal

The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis

The dynamic and urgent path of rare disease and orphan drug research

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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)

Yehoshua Berman, ... Neal J. Weinreb
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

Joe N. KornegayJoe N. Kornegay, ... Martin K. (Casey) Childers
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

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

Derralynn Hughes, Guillem Pintos-MorellGuillem Pintos-Morell
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

Anneliene Hechtelt JonkerAnneliene Hechtelt Jonker, Daniel J. O’ConnorDaniel J. O’Connor
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

Thomas Kenny, ... Deborah Woodman
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

Christine Fetro
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

Galliano Zanello, ... Virginie Hivert
Open Access Perspective DOI: 10.20517/rdodj.2023.04 24 Apr 2023
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Themed Collections
rare disease
orphan drug
Topic: Newborn Screening I - Real World Applications and Technologies
Topic: Newborn Screening II - Policy, Ethics and Patient Perspectives
Volume 3, Issue 4 (in progress)
Volume 3, Issue 3
Volume 3, Issue 2
Volume 3, Issue 1
Volume 2, Issue 4
Volume 2, Issue 3
Volume 2, Issue 2
Volume 2, Issue 1
Volume 1, Issue 4
Volume 1, Issue 3
Volume 1, Issue 2
Volume 1, Issue 1
The 2nd RDODJ Webinar
Familial Hypercholesterolemia
Special Interview with Dr. Randi J. Hagerman
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.
Special Interview with Dr. Juan Manuel Politei
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 Supports 2024 Global Rare Diseases Research Symposium
Rare Disease and Orphan Drugs Journal is delighted to announce support for the 2024 Global Rare Disease Research Symposium
Series special issues on Newborn Screening

The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis

The dynamic and urgent path of rare disease and orphan drug research

The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis
Thomas Kenny, ... Deborah Woodman
Open Access | Review | 7 Apr 2022
Views: | Downloads:
Download PDF
Connecting academia and industry for innovative drug repurposing in rare diseases: it is worth a try
Christine Fetro
Open Access | Perspective | 9 Apr 2023
Views: | Downloads:
Download PDF
Sustainable approaches for drug repurposing in rare diseases: recommendations from the IRDiRC Task Force
Galliano Zanello, ... Virginie Hivert
Open Access | Perspective | 24 Apr 2023
Views: | Downloads:
Download PDF
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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