Rare Disease and Orphan Drugs Journal
All Journals
Search
Log In
Editor-in-Chief:
Daniel Scherman
Indexing:
Dimensions
,
Lens
,
Submission to first decision:
72 days
Hot Keywords
rare disease
orphan drug
Home
About
About the Journal
Aims and Scope
Editorial Policies
Editorial Board
Junior Editorial Board
News
Partners
Advertise
Contact Us
Publish with us
For Authors
Author Instructions
Article Processing Charges
Editorial Process
Manuscript Templates
Submit a Manuscript
For Reviewers
Peer Review Guidelines
Articles
All Articles
Articles with Video Abstracts
Video Abstract Guidelines
Special Issues
All Special Issues
Ongoing Special Issues
Completed Special Issues
Closed Special Issue
Special Issue Ebooks
Special Issue Guidelines
Volumes
Pre-onlines
Features
Interviews
Webinars
Home
Articles
Special Issues
Volumes
Webinars
Videos
Menu
Rare Disease and Orphan Drugs Journal
Search
Submit
Editor-in-Chief:
Daniel Scherman
Submission to first decision:
72 days
Home
Articles
Special Issues
Volumes
Webinars
Videos
Menu
Home
Volumes
Volume 3
Volume 3, Issue 4 (2024)
Volume
Previous
Volume 3, Issue 4 (December, 2024) – 7 articles
Cover Picture:
Coming soon.
Editorial
Special issue on Fabry disease - book 1: editorial
Derralynn Hughes, Guillem Pintos-Morell
Rare Dis Orphan Drugs J
2024;3:29. DOI:
10.20517/rdodj.2024.30
Editorial | Available online: 17 Oct 2024
Views:
| Downloads:
Download PDF
A landscape map of the key global rare disease organizations
Anneliene Hechtelt Jonker
, Daniel J. O’Connor
Rare Dis Orphan Drugs J
2024;3:28. DOI:
10.20517/rdodj.2024.29
Editorial | Available online: 16 Oct 2024
Views:
| Downloads:
Download PDF
Review
First, do no harm: the role of preclinical animal models in predicting adverse events in gene therapy clinical trials for Duchenne muscular dystrophy and X-Linked myotubular myopathy
Joe N. Kornegay
, ... Martin K. (Casey) Childers
Rare Dis Orphan Drugs J
2024;3:31. DOI:
10.20517/rdodj.2024.28
Review | Available online: 3 Nov 2024
Views:
| Downloads:
Download PDF
Respiratory abnormalities in Dravet syndrome: insights from a case report and literature review
Jacopo Favaro, ... Irene Toldo
Rare Dis Orphan Drugs J
2024;3:30. DOI:
10.20517/rdodj.2023.64
Review | Available online: 21 Oct 2024
Views:
| Downloads:
Download PDF
The complexity of funding rare disease research: an IRDiRC assessment of the landscape
Lucia Monaco, ... Adam L. Hartman
Rare Dis Orphan Drugs J
2024;3:27. DOI:
10.20517/rdodj.2024.18
Review | Available online: 8 Sep 2024
Views:
| Downloads:
Download PDF
Non-viral gene therapy for neuromuscular diseases including Duchenne muscular dystrophy using nanovesicles derived from human cells
Seung Wook Oh
, ... Sung-Soo Park
Rare Dis Orphan Drugs J
2024;3:26. DOI:
10.20517/rdodj.2024.16
Review | Available online: 1 Sep 2024
Views:
| Downloads:
Download PDF
Correction
Correction: Exploratory survey study on adjunctive use of medical cannabis for Gaucher Disease (
Rare Disease and Orphan Drugs Journal
2024;
10.20517/rdodj.2024.17
)
Yehoshua Berman, ... Neal J. Weinreb
Rare Dis Orphan Drugs J
2024;3:32. DOI:
10.20517/rdodj.2024.39
Correction | Available online: 6 Nov 2024
Views:
| Downloads:
Download PDF
Actions for 0 selected articles
Download PDFs >
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
editorialoffice@rdodjournal.com
Navigation
Contact Us
Sitemap
Follow Us
Twitter
LinkedIn
Navigation
Contact Us
Sitemap
Committee on Publication Ethics
https://publicationethics.org/members/rare-disease-and-orphan-drugs-journal
Portico
All published articles are preserved here permanently:
https://www.portico.org/publishers/oae/
Committee on Publication Ethics
https://publicationethics.org/members/rare-disease-and-orphan-drugs-journal
Portico
All published articles are preserved here permanently:
https://www.portico.org/publishers/oae/