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Volume 5
Volume 5, Issue 3 (2026)
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Volume 5, Issue 2
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Volume 5, Issue 3 (2026) – 10 articles
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Review
Diagnosis and therapy of Niemann-Pick disease type C1- what have we learned that can help veterinary patients?
Charles H. Vite, ... Margret L. Casal
Rare Dis Orphan Drugs J
. 2026;5:23.
DOI:
10.20517/rdodj.2025.74
Review | Available online: 2 Jul 2026
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Immunotherapeutic advances for the treatment of osteosarcoma, driven by studies in pet dogs with spontaneous disease
Grace L. Edmunds
, ... Nicola J. Mason
Rare Dis Orphan Drugs J
. 2026;5:25.
DOI:
10.20517/rdodj.2026.12
Review | Available online: 9 Jul 2026
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Advances in the application of deep learning in computed tomography imaging analysis of rare lung diseases
Bingyi Liu, ... Min Xie
Rare Dis Orphan Drugs J
. 2026;5:26.
DOI:
10.20517/rdodj.2025.88
Review | Available online: 20 Jul 2026
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Original Article
Computational modelling of cerebrospinal fluid flow in a translational canine model
Ryan Jones
, ... Srdjan Cirovic
Rare Dis Orphan Drugs J
. 2026;5:24.
DOI:
10.20517/rdodj.2026.05
Original Article | Available online: 9 Jul 2026
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Case Report
Primary sclerosing encapsulating peritonitis with cryptorchidism in an older man: case report and literature review
Yeqing Chen, ... Hongyu Zhou
Rare Dis Orphan Drugs J
. 2026;5:22.
DOI:
10.20517/rdodj.2025.79
Case Report | Available online: 1 Jul 2026
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Multidisciplinary management of concurrent hypogonadism, diabetes, and hypothyroidism in Woodhouse-Sakati syndrome: a case report and preliminary clinical management experience
Xiaoyang Shi, ... Huijuan Yuan
Rare Dis Orphan Drugs J
. 2026;5:29.
DOI:
10.20517/rdodj.2026.31
Case Report | Available online: 3 Sep 2026
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Commentary
Transcriptional adaptation opens new therapeutic possibilities for Duchenne muscular dystrophy and rare neuromuscular diseases
Anika Nusrat
, ... Xander H. T. Wehrens
Rare Dis Orphan Drugs J
. 2026;5:27.
DOI:
10.20517/rdodj.2026.35
Commentary | Available online: 13 Aug 2026
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AAV9.LAMP2B
gene therapy for Danon disease: from early clinical experience to evolving safety and trial design considerations
Alessia Argirò
, ... Iacopo Olivotto
Rare Dis Orphan Drugs J
. 2026;5:30.
DOI:
10.20517/rdodj.2026.26
Commentary | Available online: 27 Sep 2026
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Receptor-mediated brain delivery in MPS II: a platform turning point for rare disease therapeutics
Betul Celik, ... Shunji Tomatsu
Rare Dis Orphan Drugs J
. 2026;5:31.
DOI:
10.20517/rdodj.2026.20
Commentary | Available online: 27 Sep 2026
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Perspective
Beyond copper retention: a dynamic flux-compartment-response model of Wilson disease
Wei Hou
, ... Sujun Zheng
Rare Dis Orphan Drugs J
. 2026;5:28.
DOI:
10.20517/rdodj.2026.36
Perspective | Available online: 17 Aug 2026
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Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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Committee on Publication Ethics
https://members.publicationethics.org/members/rare-disease-and-orphan-drugs-journal
Portico
All published articles are preserved here permanently:
https://www.portico.org/publishers/oae/