Exploring Novel Molecular Pathways and Emerging Treatments for Neuromuscular Disorders
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Medicine".
Deadline for manuscript submissions: 31 July 2025 | Viewed by 267
Special Issue Editor
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 witnessing an exciting era in the field of neuromuscular diseases, with several promising therapeutic approaches progressing through clinical trials and, in some cases, achieving regulatory approval. Disorders, such as spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS), have seen notable advances, with the development of disease-modifying therapies that target specific molecular mechanisms. Nevertheless, for many neuromuscular conditions, effective treatments remain elusive, and therapeutic strategies often focus on a limited set of biological targets.
Recent progress in elucidating novel molecular and cellular underpinnings of these diseases has expanded the landscape of potential therapeutic interventions, providing renewed hope for patients and their families.
This Special Issue of Biomolecules will focus on the latest research and advances in the development of treatments for neuromuscular disorders. We aim to showcase innovative molecular and biochemical approaches that drive the discovery of new therapeutic strategies. Topics of interest include the identification of novel molecular targets, disease mechanisms, and drug candidates, as well as the exploration of combinatorial therapies that integrate complementary mechanisms of action to enhance treatment efficacy.
We welcome contributions that explore the molecular pathways involved in neuromuscular diseases, the rational design of therapeutic compounds, and proof-of-concept studies highlighting the translational potential of emerging treatments. Both original research and comprehensive review articles are encouraged, as we seek to provide a broad and insightful overview of the current state and future directions in the quest to develop effective therapies for neuromuscular disorders.
Dr. Arturo Lopez-Castel
Guest Editor
Manuscript Submission Information
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Keywords
- gene therapy
- repurposing
- therapeutic oligonucleotides
- personalized medicine
- biomarkers
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