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 12 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 11 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 5 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 2 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 1 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 8 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 28 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 30 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)
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Volume 3, Issue 4
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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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