Exploring Novel Molecular Pathways and Emerging Treatments for Myotonic Dystrophy

A special issue of Biomolecules (ISSN 2218-273X).

Deadline for manuscript submissions: 15 February 2025

Special Issue Editor


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Guest Editor
1. Human Translational Genomics Group, BIOTECMED Research Institute, Faculty of Biology, University of Valencia, Valencia, Spain
2. INCLIVA Biomedical Research Institute, Valencia, Spain
Interests: rare diseases; drug development; personalized medicine
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

We are currently living through an exciting time in the myotonic dystrophy (DM1 and DM2) field, with the first consolidated molecule developments moving through clinical trials. However, despite significant research efforts, no disease-modifying treatments are currently available for DM patients, and approaches are still limited to a few biological targets. Recent advances in understanding the molecular mechanisms underlying these diseases have opened new avenues for therapeutic intervention, offering hope for improved patient outcomes.

This Special Issue of Biomolecules will focus on the latest research and developments in the search for treatments for DM1 and DM2. We aim to present cutting-edge molecular and biochemical insights for the development of novel therapeutic strategies. Topics will include the identification and development of novel molecular targets and mechanisms connected to DM dysregulation and the identification of unique drug candidates. Additionally, we are open to exploring how combinatorial therapies around the leveraging of complementary mechanisms of action will enhance DM patients´ treatment efficacy.

We invite contributions that delve into the molecular pathways involved in DM1 and DM2, the design of therapeutic molecules, and proof-of-concept studies demonstrating the potential of new treatments. Both research and review articles are welcome, as we seek to compile a comprehensive overview of the current state and future directions in the fight against myotonic dystrophies.

Dr. Arturo Lopez-Castel
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. Biomolecules is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2700 CHF (Swiss Francs). 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

  • gene therapy
  • repurposing
  • therapeutic oligonucleotides
  • personalized medicine
  • biomarkers

Published Papers

This special issue is now open for submission.
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