Editor-in-Chief: Daniel Scherman
Submission to first decision: 72 days

Rare Disease and Orphan Drugs Journal

Articles

Most Recent | Most Viewed | Most Downloaded

The genotype and phenotype correlation of Prader-Willi syndrome

Open Access Review DOI: 10.20517/rdodj.2024.20 26 Nov 2024
Views: Downloads:
Download PDF

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)

Open Access Correction DOI: 10.20517/rdodj.2024.39 6 Nov 2024
Views: Downloads:
Download PDF

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

Open Access Review DOI: 10.20517/rdodj.2024.28 3 Nov 2024
Views: Downloads:
Download PDF

Respiratory abnormalities in Dravet syndrome: insights from a case report and literature review

Open Access Review DOI: 10.20517/rdodj.2023.64 21 Oct 2024
Views: Downloads:
Download PDF

Special issue on Fabry disease - book 1: editorial

Open Access Editorial DOI: 10.20517/rdodj.2024.30 17 Oct 2024
Views: Downloads:
Download PDF

The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis

Open Access Review DOI: 10.20517/rdodj.2022.04 7 Apr 2022
Views: Downloads:
Download PDF

Connecting academia and industry for innovative drug repurposing in rare diseases: it is worth a try

Open Access Perspective DOI: 10.20517/rdodj.2023.06 9 Apr 2023
Views: Downloads:
Download PDF

Sustainable approaches for drug repurposing in rare diseases: recommendations from the IRDiRC Task Force

Open Access Perspective DOI: 10.20517/rdodj.2023.04 24 Apr 2023
Views: Downloads:
Download PDF
Familial Hypercholesterolemia
During the interview, Dr. Hagerman elaborated on the key points of her article, current research, and prospects within the field of Fragile X Syndrome, and offered suggestions for the future development of the journal.
During the interview, Dr. Politei talked about the highlight of this article, his current research, prospects in the field of Fabry diseases, suggestions for the future development of the journal.
Rare Disease and Orphan Drugs Journal is delighted to announce support for the 2024 Global Rare Disease Research Symposium
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
Follow Us

Portico

All published articles are preserved here permanently:

https://www.portico.org/publishers/oae/

Portico

All published articles are preserved here permanently:

https://www.portico.org/publishers/oae/