Rare Disease and Orphan Drugs Journal
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Editor-in-Chief: Daniel Scherman
Indexing: ESCI, Scopus, Dimensions, Lens, ASCI,
Median time to first editorial decision: 7 days
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Rare Disease and Orphan Drugs Journal
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Editor-in-Chief: Daniel Scherman
Median time to first editorial decision: 7 days
Home Articles Special Topics 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

Gut microbial dysbiosis in Gaucher disease: implications for associated complications

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Commentary on: ribozyme-activated RNA trans-ligation: an emerging strategy for large gene delivery in muscular dystrophies

Martin K. Childers, ...Hichem Tasfaout
Open Access Commentary DOI: 10.20517/rdodj.2025.35 13 Mar 2026
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Commentary on gait analysis in Duchenne muscular dystrophy: integrating contracture, strength, and kinematic perspectives

Daniel Scherman
Open Access Commentary DOI: 10.20517/rdodj.2025.58 12 Mar 2026
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The critical importance of recognizing Gaucher disease in the fifth decade and beyond

Daniel Scherman
Open Access Editorial DOI: 10.20517/rdodj.2026.02 6 Mar 2026
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Adeno-associated viral vector gene therapy for canine hemophilia

Bhavya S. Doshi, ...Mary Beth Callan
Open Access Review DOI: 10.20517/rdodj.2025.66 3 Mar 2026
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Plexiform neurofibromas in neurofibromatosis type 1: current and emergent therapeutic strategies

Hannah Kim, ...Gurcharanjeet Kaur
Open Access Perspective DOI: 10.20517/rdodj.2025.52 2 Mar 2026
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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 9 Sep 2024
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Newborn screening in South Africa: the past, present, and plans for the future

Helen L. Malherbe, ... Barend C. Vorster
Open Access Commentary DOI: 10.20517/rdodj.2023.49 29 Feb 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 31 Jul 2024
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Themed Collections
rare disease
orphan drug
Topic: Duchenne Muscular Dystrophy (DMD): Progress in Research and Insights
Topic: Advancements in Diagnosis and Treatment of Familial Hypercholesterolemia
Topic: Systemic Lupus Erythematosus: Early Diagnosis and Management Strategies
Volume 5, Issue 1 (in progress)
Volume 4, Issue 4
Volume 4, Issue 3
Volume 4, Issue 2
Volume 4, Issue 1
Volume 3, Issue 4
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
Rare Disease and Orphan Drugs Journal Reports Key Milestones for 2025
Journal sees increased submissions, broader international authorship, and anticipates first Impact Factor in 2026
Rare Disease and Orphan Drugs Journal Accepted for Inclusion in Emerging Sources Citation Index (ESCI)
Rare Disease and Orphan Drugs Journal (RDODJ) has been officially indexed in the Emerging Sources Citation Index (ESCI) of Web of Science as of September 16, 2025!
Congratulations!
Fifteen Editorial Board Members of RDODJ Named among 2025 World's Top 2% of Scientists!
Scopus Indexing Achieved: A New Chapter for RDODJ!
Starting from 2022, all publications of RDODJ will be fully indexed in Scopus.
The 2nd RDODJ Webinar
Familial Hypercholesterolemia

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

Gut microbial dysbiosis in Gaucher disease: implications for associated complications

The complexity of funding rare disease research: an IRDiRC assessment of the landscape
Lucia Monaco, ... Adam L. HartmanAdam L. Hartman
Open Access | Review | 9 Sep 2024
Views: | Downloads:
Download PDF
Newborn screening in South Africa: the past, present, and plans for the future
Helen L. Malherbe, ... Barend C. Vorster
Open Access | Commentary | 29 Feb 2024
Views: | Downloads:
Download PDF
Framing the European Rare Diseases field through a structured movement of patient organisations
Maria Cavaller-BellaubiMaria Cavaller-Bellaubi, ... Yann Le Cam
Open Access | Perspective | 31 Jul 2024
Views: | Downloads:
Download PDF

Coming soon.

Volume 5, Issue 1 (in progress)
Volume 4, Issue 4
Volume 4, Issue 3
Volume 4, Issue 2
Volume 4, Issue 1
Volume 3, Issue 4
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
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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