Rare Disease and Orphan Drugs Journal
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Editor-in-Chief: Daniel Scherman
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Submission to first decision: 72 days
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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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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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The European joint programme on rare diseases: building the rare diseases research ecosystem

Yanis Mimouni, ... Daria JulkowskaDaria Julkowska
Open Access Perspective DOI: 10.20517/rdodj.2024.06 23 Jun 2024
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Newborn screening in Mexico and Latin America: present and future

Claudia Gonzaga-JaureguiClaudia Gonzaga-Jauregui, ... Juana Inés Navarrete-Martínez
Open Access Perspective DOI: 10.20517/rdodj.2024.02 23 Jun 2024
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The division of rare diseases research innovation at the national center for advancing translational sciences, NIH: mission, history, and current research activities

P. J. Brooks, ... Tiina UrvTiina Urv
Open Access Perspective DOI: 10.20517/rdodj.2023.27 2 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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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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Enzyme replacement therapy: current challenges and drug delivery prospects via extracellular vesicles

Amanda K. A. SilvaAmanda K. A. Silva, ... Ibane AbasoloIbane Abasolo
Open Access Editorial DOI: 10.20517/rdodj.2022.09 3 Jul 2022
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Topic: Latest Advances in Primary Immunodeficiency Diseases

Prof. Siobhan Burns

Submission Deadline: 31 Dec 2024

Published articles: 0

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

Prof. Maurizio Delvecchio, PhD; Prof. Phillip D.K. Lee, MD

Submission Deadline: 15 Nov 2024

Published articles: 0

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rare disease
orphan drug
Volume 3, Issue 3 (in progress)
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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
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
Enzyme replacement therapy: current challenges and drug delivery prospects via extracellular vesicles
Amanda K. A. SilvaAmanda K. A. Silva, ... Ibane AbasoloIbane Abasolo
Open Access | Editorial | 3 Jul 2022
Views: | Downloads:
Download PDF

Topic: Latest Advances in Primary Immunodeficiency Diseases

Prof. Siobhan Burns

Submission Deadline: 31 Dec 2024

Published articles: 0

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

Prof. Maurizio Delvecchio, PhD; Prof. Phillip D.K. Lee, MD

Submission Deadline: 15 Nov 2024

Published articles: 0

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