The Rare Disease and Orphan Drugs Journal (RDODJ) is an international, peer-reviewed, open access journal for the publication of innovative research works on various aspects of this rapidly growing multidisciplinary and interdisciplinary field.
RDODJ will report on scientific advances in ;the genetics of rare diseases, the molecular basis of the pathologies, and translational research on diagnosis, prevention and treatment.
In addition, RDODJ aims to provide a forum for scientific studies and discussion covering the important regulatory, socio-economic and human science issues related to rare diseases and orphan drugs.
The ultimate objective of RDODJ is to promote the dissemination of research results and scientific discussion among the research community, practitioners, and patient-advocacy organizations.
The scope of RDODJ covers the following research topics:
Genetics of rare diseases. Non-genetic rare diseases, rare cancers, tropical rare diseases
Diagnosis and undiagnosed rare diseases
Epidemiology, registries, data bases, and artificial intelligence
Pathophysiological mechanism. Biochemistry and cell biology
Protein, gene, and cell therapy. Precision medicine and genome editing
Drug repurposing and delivery
Preclinical translational research. Clinical research and methodology
Regulatory and practice guidelines
Medico-economic, health policies and services organization and health promotion
Social sciences: patients' needs, quality of life.