Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene. Recent studies have focused on various therapeutic approaches to mitigate disease progression. The EMBARK trial explored the effects of delandistrogene moxeparvovec, a gene transfer therapy, demonstrating stabilization or reduced progression of muscle pathology as measured by quantitative magnetic resonance imaging (ref: Vandenborne doi.org/10.1001/jamaneurol.2025.0992/). Additionally, the use of DG9, a cell-penetrating peptide, enhanced the nuclear uptake of phosphorodiamidate morpholino oligomers (PMOs), promoting exon skipping and restoring muscle function in DMD models (ref: Shah doi.org/10.1038/s41467-025-59494-8/). Another promising approach involved repurposing dimethyl fumarate, which showed significant reductions in muscle inflammation and fibrosis in an aggravated mdx mouse model, suggesting its potential as a therapeutic agent (ref: Kourakis doi.org/10.1016/j.redox.2025.103676/). Moreover, innovative strategies such as polymer-based coatings for adeno-associated viral particles have been proposed to evade immune responses, enhancing the efficacy of gene therapies (ref: Pinto doi.org/10.1016/j.jconrel.2025.113896/). Forskolin treatment has also been shown to improve muscle regeneration and reduce fibrosis in DMD, although its long-term therapeutic potential remains to be fully evaluated (ref: Cojocaru doi.org/10.1186/s13395-025-00381-7/). Collectively, these studies highlight the multifaceted approaches being explored to combat DMD, emphasizing the need for continued research to optimize treatment strategies.

Amyotrophic Lateral Sclerosis (ALS) remains a challenging neurodegenerative disease with limited therapeutic options. Recent clinical trials have investigated novel treatments aimed at improving patient outcomes. The MIROCALS trial assessed the efficacy and safety of low-dose interleukin-2 (IL-2) as an adjunct therapy to riluzole, revealing promising results in enhancing immune modulation in ALS patients (ref: Bensimon doi.org/10.1016/S0140-6736(25)00262-4/). In another significant study, the HEALEY ALS Platform Trial evaluated trehalose, a disaccharide that activates autophagy, but reported serious adverse events, highlighting the complexities of ALS treatment development (ref: doi.org/10.1016/S1474-4422(25)00173-5/). These findings underscore the necessity for careful patient selection and monitoring in clinical trials, as well as the importance of understanding the underlying mechanisms of ALS to develop effective therapies. The contrasting results from these trials reflect the ongoing challenges in ALS research, emphasizing the need for innovative approaches and combination therapies to address this devastating condition.

Idiopathic inflammatory myopathies (IIM) encompass a group of autoimmune disorders characterized by muscle inflammation and weakness. Recent studies have focused on understanding the long-term outcomes and treatment responses in these patients. A large-scale longitudinal cohort study revealed that patients with anti-MDA5-positive dermatomyositis exhibited higher standardized mortality ratios compared to other IIM serotypes, with a cumulative disease-free survival rate of only 29.3% at ten years (ref: Chen doi.org/10.1016/j.jaut.2025.103435/). Furthermore, the characterization of local antibody responses in inclusion body myositis patients has provided insights into the disease's pathogenesis, suggesting a potential role for autoantibodies in muscle damage (ref: Jayaraman doi.org/10.1016/j.jaut.2025.103437/). In addition, the safety and efficacy of ultrasound-guided needle muscle biopsy have been evaluated, demonstrating its utility in diagnosing IIM with a high success rate (ref: Costa doi.org/10.1093/rheumatology/). These findings highlight the importance of early diagnosis and tailored treatment strategies in managing IIM, as well as the need for further research into novel therapeutic options such as immunoadsorption, which showed moderate to major improvement in refractory cases (ref: Kastrati doi.org/10.1093/rheumatology/).

Myopathies and muscle disorders represent a diverse group of conditions with varying genetic and phenotypic presentations. Recent research has focused on understanding the underlying mechanisms and potential therapeutic strategies. A study on negamycin, an antibiotic with readthrough-promoting activity, suggests its potential as a therapeutic agent for DMD and other hereditary diseases, highlighting its unique properties (ref: Wang doi.org/10.1038/s41589-025-01898-0/). Additionally, research into dynamin 2 mutations has revealed opposing effects on muscle phenotypes, indicating the complexity of genetic contributions to myopathies (ref: Goret doi.org/10.1038/s41467-025-59925-6/). The development of template-assisted sequence knockin (TASK) strategies has shown promise in restoring dystrophin function in DMD models, emphasizing the need for targeted genetic therapies (ref: Fatehi doi.org/10.1016/j.ymthe.2025.05.005/). Furthermore, the identification of NOTCH2 mutations in centronuclear myopathy has provided insights into the genetic basis of this condition, underscoring the importance of genetic screening in myopathy diagnosis (ref: Lin doi.org/10.1016/j.ymthe.2025.04.041/). Collectively, these studies illustrate the ongoing efforts to unravel the complexities of myopathies and develop effective therapeutic interventions.

The genetic and molecular mechanisms underlying myopathies are critical for understanding disease pathogenesis and developing targeted therapies. Recent studies have highlighted the role of DUX4 in facioscapulohumeral muscular dystrophy (FSHD), revealing its involvement in regulating gene expression and chromatin accessibility (ref: Zheng doi.org/10.1126/sciadv.adt5356/). Additionally, novel LMNA variants associated with familial partial lipodystrophy have been characterized, providing insights into genotype-phenotype relationships in this rare disorder (ref: Anum doi.org/10.1210/clinem/). The integration of single-cell functional-proteomic profiling has unveiled shifts in myofibre specificity in nemaline myopathy, emphasizing the need for advanced methodologies to dissect muscle biology (ref: Seaborne doi.org/10.1113/JP288363/). Furthermore, the identification of new SCN4A variants linked to myotonic disorders underscores the genetic heterogeneity present in myopathies (ref: Periviita doi.org/10.1111/ene.70157/). These findings collectively enhance our understanding of the molecular underpinnings of myopathies and highlight the potential for precision medicine approaches in their management.

Therapeutic approaches for muscle disorders are rapidly evolving, with recent clinical trials focusing on optimizing treatment strategies. The development of recommendations for treating juvenile dermatomyositis (JDM) has emerged from a systematic literature review, aiming to improve patient outcomes through targeted therapies (ref: Ravelli doi.org/10.1016/j.ard.2025.04.024/). Additionally, the repurposing of dimethyl fumarate has shown promise in alleviating muscle inflammation in DMD models, suggesting its potential as a viable treatment option (ref: Kourakis doi.org/10.1016/j.redox.2025.103676/). The exploration of metabolic factors in Parkinson's disease has revealed the impact of dietary interventions on muscle health, emphasizing the importance of a holistic approach to treatment (ref: de Mello doi.org/10.1016/j.molmet.2025.102163/). Furthermore, innovative neuromodulation strategies are being investigated for fibromyalgia, aiming to address central sensitization symptoms (ref: Or-Borichev doi.org/10.1186/s12916-025-04138-3/). These studies reflect the dynamic landscape of therapeutic research in muscle disorders, highlighting the need for continued innovation and patient-centered approaches.

Chronic Fatigue Syndrome (CFS) and fibromyalgia (FM) are complex conditions that have garnered increasing research attention. Recent findings suggest that human endogenous retrovirus (HERV) activation may serve as a distinguishing biomarker between ME/CFS and FM, potentially aiding in diagnosis and understanding of these syndromes (ref: Giménez-Orenga doi.org/10.7554/eLife.104441/). The impact of the COVID-19 pandemic on public perceptions of ME/CFS has also been explored, revealing a shift in attitudes towards the condition following updated clinical guidelines that emphasize its biomedical nature (ref: Khakban doi.org/10.2196/65087/). Additionally, novel neuromodulation techniques targeting limbic dysregulation are being investigated for their potential to alleviate symptoms associated with FM, highlighting the need for innovative treatment strategies (ref: Or-Borichev doi.org/10.1186/s12916-025-04138-3/). These studies underscore the importance of understanding the underlying mechanisms of CFS and FM, as well as the need for continued advocacy and research to improve patient outcomes.

Muscle regeneration and repair mechanisms are critical areas of research, particularly in the context of muscular dystrophies. Recent studies have investigated the role of the PIEZO1 channel in myogenic differentiation, demonstrating that inhibition of this channel promotes muscle repair and reduces damage in DMD models (ref: Wang doi.org/10.1186/s13395-025-00383-5/). Furthermore, research into the glucocorticoid axis has revealed that muscle wasting in lung-injured mice is not primarily driven by glucocorticoid receptor signaling, suggesting alternative pathways may be involved in muscle atrophy (ref: Chuang Key doi.org/10.1152/ajpendo.00039.2025/). These findings highlight the complexity of muscle regeneration processes and the need for targeted interventions to enhance muscle repair. Collectively, these studies contribute to a deeper understanding of the cellular and molecular mechanisms governing muscle regeneration, paving the way for innovative therapeutic strategies.