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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The complexity of funding rare disease research: an IRDiRC assessment of the landscape

Lucia Monaco, ... Adam L. HartmanAdam L. Hartman
Open Access Review DOI: 10.20517/rdodj.2024.18 8 Sep 2024
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Non-viral gene therapy for neuromuscular diseases including Duchenne muscular dystrophy using nanovesicles derived from human cells

Seung Wook OhSeung Wook Oh, ... Sung-Soo Park
Open Access Review DOI: 10.20517/rdodj.2024.16 1 Sep 2024
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Exploratory survey study on adjunctive use of medical cannabis for Gaucher disease

Yehoshua Berman, ... Neal J. Weinreb
Open Access Original Article DOI: 10.20517/rdodj.2024.17 22 Aug 2024
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Patient organizations: advocating for timely newborn screening & improved quality of life

Helen L. Malherbe, ... Chiuhui Mary WangChiuhui Mary Wang
Open Access Perspective DOI: 10.20517/rdodj.2024.11 14 Aug 2024
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Framing the European Rare Diseases field through a structured movement of patient organisations

Maria Cavaller-BellaubiMaria Cavaller-Bellaubi, ... Yann Le Cam
Open Access Perspective DOI: 10.20517/rdodj.2023.58 30 Jul 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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Topic: Advances in Neurofibromatosis - The Future is Bright

Prof. Allan E. Rubenstein; Gurcharanjeet Kaur, MD

Submission Deadline: 28 Feb 2025

Published articles: 0

Topic: Thalassemia: Progress in Research and Insights

Prof. Liye Yang

Submission Deadline: 15 Jan 2025

Published articles: 0

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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 1st RDODJ Ideological Collision Webinar
Duchenne Muscular Dystrophy
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

Topic: Advances in Neurofibromatosis - The Future is Bright

Prof. Allan E. Rubenstein; Gurcharanjeet Kaur, MD

Submission Deadline: 28 Feb 2025

Published articles: 0

Topic: Thalassemia: Progress in Research and Insights

Prof. Liye Yang

Submission Deadline: 15 Jan 2025

Published articles: 0

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