Neuromuscular disorders: biomarkers, precision diagnosis, and targeted therapeutics

In the rapidly evolving field of neuromuscular disorders (NMDs), ongoing research is crucial to develop precise diagnostic tools and targeted therapies. This Research Topic addresses the urgent need for biomarker discovery in conditions such as Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and amyotrophic lateral sclerosis (ALS) among others. Despite advancements, crucial challenges remain in identifying reliable biomarkers that can guide early diagnosis, monitor progression, and tailor therapeutic strategies to individual patient profiles. Recent studies have started to unveil potential biomarkers across various biological sources, including blood, cerebrospinal fluid, and exosomes, employing advanced proteomic and genomic technologies. Nevertheless, the comprehensive utilization of these markers in clinical practice is still underexplored, with a need for integration into real-world applications to improve outcomes.

This Research Topic aims to foster innovative approaches in the diagnosis and management of NMDs through precision medicine. We seek research that not only explores novel biomarkers but also tests these markers in clinical settings to establish effective, personalized treatment regimens. Our goal is both to enhance understanding of the molecular basis of NMDs and to translate these findings into actionable, patient-specific therapies.

To gather further insights, this topic is focused on enhancing the practical and clinical aspects of neuromuscular disorders. Initially, research within this scope aims at refining diagnostic accuracy and therapeutic targeting according to emerging biomarkers. We welcome articles addressing, but not limited to, the following themes:

• Emerging biomarkers for early diagnosis and disease monitoring;
• Biomarkers for disease stratification and clinical management;
• Gene editing and targeted therapies for neuromuscular diseases;
• Immunomodulatory approaches in neuromuscular disorders;
• Molecular pathways in neuromuscular diseases and therapeutic targeting;
• Biomarker validation and clinical implementation;
• Patient-centered approaches: bridging the gap between biomarker discovery and clinical application.

Keywords: Biomarker Discovery, Neuromuscular Disorders, Precision Medicine, Targeted Therapeutics, Gene Editing and RNA Therapies

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.