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Biomolecules
Special Issues
Exploring Novel Molecular Pathways and Emerging Treatments for Neuromuscular Disorders
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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: 28 February 2026 | Viewed by 424

Special Issue Editor

Dr. Arturo Lopez-Castel

E-Mail Website
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 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

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 250 words) can be sent to the Editorial Office for assessment.

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

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Published Papers (1 paper)

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Research

18 pages, 6073 KB  
Article
Absence of Neuromuscular Dysfunction in Mice with Gut Epithelium-Restricted Expression of ALS Mutation hSOD1G93A
by Li Dong, Xuejun Li, Ang Li, Jianxun Yi, Yanan Vockery, Yan Chang, Zui Pan, Marco Brotto and Jingsong Zhou
Biomolecules 2026, 16(2), 253; https://doi.org/10.3390/biom16020253 - 5 Feb 2026
Abstract
Amyotrophic Lateral Sclerosis (ALS) is a devastating neuromuscular disorder characterized by the progressive loss of motor neurons and skeletal muscle, ultimately leading to respiratory failure and death, typically within 3–5 years following diagnosis. While the death of motor neurons is the pathological hallmark, [...] Read more.
Amyotrophic Lateral Sclerosis (ALS) is a devastating neuromuscular disorder characterized by the progressive loss of motor neurons and skeletal muscle, ultimately leading to respiratory failure and death, typically within 3–5 years following diagnosis. While the death of motor neurons is the pathological hallmark, ALS is increasingly recognized as a systemic disorder involving non-motor systems. Gastrointestinal dysfunction has been widely observed in both ALS patients and animal models. However, because gut abnormalities and neuromuscular degeneration are intertwined during ALS disease progression, it remains unclear whether these gut abnormalities are merely a consequence of neuromuscular degeneration or whether they play a crucial role in initiating it. In this study, we investigated whether an ALS-associated mutation expressed exclusively in the gut can directly affect neuromuscular function. We generated a novel transgenic mouse model, Gut-hG93A, which overexpresses the human ALS mutation hSOD1G93A specifically in the epithelial cells of the intestine at a level comparable to the endogenous mouse SOD1. We found that the specific overexpression of hSOD1G93A in gut epithelial cells did not cause abnormalities in the structure of the tight junctions or in gut permeability. Furthermore, there were no significant differences between Gut-hG93A and control mice regarding lifespan, body weight, or neuromuscular activities, including grip strength, daily travel distance and in vivo muscle contractility. These findings suggest that the ALS-associated hSOD1G93A mutation, when expressed solely in the gut epithelium, is not sufficient to initiate neuromuscular degeneration of systemic ALS-like pathology. Full article
(This article belongs to the Special Issue Exploring Novel Molecular Pathways and Emerging Treatments for Neuromuscular Disorders)
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