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

Articles

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Fabry nephropathy: a treatable cause of chronic kidney disease

Michael L. WestMichael L. West, ... Daniel G. Bichet
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

Claudia Di Berardino, ... Gaia Colasante
Open Access Review DOI: 10.20517/rdodj.2024.07 8 Jul 2024
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Neuropathy and pain in Fabry disease

Vijay Krishna Medala, Nurcan ÜçeylerNurcan Üçeyler
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

Elena-Alexandra TãtaruElena-Alexandra Tãtaru, ... David A. PearceDavid A. Pearce
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

Christiane Auray-BlaisChristiane Auray-Blais, ... Jeffrey A. Medin
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

Christine Fetro
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

Thomas Kenny, ... Deborah Woodman
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

Galliano Zanello, ... Virginie Hivert
Open Access Perspective DOI: 10.20517/rdodj.2023.04 24 Apr 2023
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Topic: Latest Advances in Primary Immunodeficiency Diseases

Prof. Siobhan Burns

Submission Deadline: 31 Dec 2024

Published articles: 0

Topic: Advances in Mucopolysaccharidosis and Alpha Mannosidosis

Dr. Karolina Stepien; Dr. Iain Hargreaves

Submission Deadline: 31 Dec 2024

Published articles: 0

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rare disease
orphan drug
Volume 3, Issue 3 (in progress)
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Volume 2, Issue 1
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Volume 1, Issue 1
Introducing our new preprint platform, a fast and efficient way to share your research with the academic community.
We consistently welcome suggestions for improvements from both readers and authors (send an e-mail to preprint@scierxiv.com).
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

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
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
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

Topic: Latest Advances in Primary Immunodeficiency Diseases

Prof. Siobhan Burns

Submission Deadline: 31 Dec 2024

Published articles: 0

Topic: Advances in Mucopolysaccharidosis and Alpha Mannosidosis

Dr. Karolina Stepien; Dr. Iain Hargreaves

Submission Deadline: 31 Dec 2024

Published articles: 0

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