Drug Repurposing in Rare Diseases: From Molecular Insights to Patient Benefits
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pharmacology".
Deadline for manuscript submissions: 30 November 2024 | Viewed by 401
Special Issue Editor
Interests: drug repurposing; rare diseases; patient engagement; patient-centric approaches; translational research; biomedical innovation; molecular genetics; disease mechanisms and molecular dysfunctions
Special Issue Information
Dear Colleagues,
Of the more than 7,000 rare diseases currently identified, less than 6% currently have an approved treatment option, despite approximately 80% having a known genetic origin or molecular basis, highlighting the tremendous unmet needs in drug development.
In response to these challenges, drug repurposing has emerged as a promising strategy to address therapeutic gaps and have a meaningful impact on the lives of patients suffering from rare diseases. We are pleased to invite you to contribute to our upcoming Special Issue entitled “Drug Repurposing in Rare Diseases: From Molecular Insights to Patient Benefits”. This Special Issue aims to explore the innovative potential of drug repurposing strategies, specifically within the context of rare diseases, charting the course from molecular insights to application and tangible patient benefits. By investigating the molecular mechanisms underlying these conditions, we seek to uncover novel therapeutic opportunities. Emphasizing patient-centric approaches, our goal is to illuminate how these insights can pave the way for novel treatments that address the unmet needs of patients suffering from rare diseases.
We encourage submissions that delve into molecular mechanisms underlying rare diseases and propose novel repurposing strategies.
Key themes for contributions include but are not limited to:
- Case studies showcasing successful drug repurposing efforts;
- Molecular insights driving the identification and validation of repurposing opportunities;
- Preclinical studies evaluating repurposed drugs for rare diseases.
Additionally, we welcome contributions on other relevant themes that address critical aspects of drug repurposing and its application to rare diseases. Please note that submissions should integrate biomolecular experimentation/considerations to align with the scope of our journal.
Your expertise and contributions are essential in improving treatment options for the rare disease community. Join us in illuminating new therapeutic pathways and fostering collaboration in this vital area of research.
Dr. Claudia Fuchs
Guest Editor
Manuscript Submission Information
Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.
Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.
Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.
Keywords
- rare diseases/rare genetic diseases
- drug repurposing/repositioning
- molecular insights
- shared molecular pathways
- preclinical trials
- case studies
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