Rare Disease and Orphan Drugs Journal
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Editor-in-Chief:
Daniel Scherman
Submission to first decision:
72 days
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Volume 4
Volume 4, Issue 1 (2025)
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Volume 4, Issue 1 (2025) – 5 articles
Cover Picture:
Coming soon.
Review
The necessity for skeletal muscle contractile assays to assess treatment efficacy in DMD
Claire Yuan, ... Robert W. Grange
Rare Dis Orphan Drugs J
. 2025;4:5.
DOI:
10.20517/rdodj.2024.34
Review | Available online: 4 Mar 2025
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Organellar crosstalk as a potential therapeutic target for rare neurodegenerative diseases
Josephine J. Lam, ... Reena V. Kartha
Rare Dis Orphan Drugs J
. 2025;4:4.
DOI:
10.20517/rdodj.2024.55
Review | Available online: 27 Feb 2025
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Fabry disease in women: beyond the role of “carriers”
Miguel-Ángel Barba-Romero, Rosario Sánchez-Martínez
Rare Dis Orphan Drugs J
. 2025;4:2.
DOI:
10.20517/rdodj.2024.45
Review | Available online: 24 Feb 2025
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Original Article
Long-term impact of corticosteroid therapy on adult height in juvenile myasthenia gravis patients: a retrospective multicenter cohort study
Ying Tan
, ... Yun Jing
Rare Dis Orphan Drugs J
. 2025;4:3.
DOI:
10.20517/rdodj.2024.36
Original Article | Available online: 25 Feb 2025
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Electroencephalogram (EEG) network-level excitation-inhibition in
GRIN2B
-related neurodevelopmental disorders: a pilot case-control series
Jennifer R. Ramautar
, ... Hilgo Bruining
Rare Dis Orphan Drugs J
. 2025;4:1.
DOI:
10.20517/rdodj.2024.27
Original Article | Available online: 9 Jan 2025
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Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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Committee on Publication Ethics
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Portico
All published articles are preserved here permanently:
https://www.portico.org/publishers/oae/