High-resolution neuromuscular ultrasound (US) is a valuable tool for the localization, diagnosis, and provision of conservative treatment to peripheral entrapment mononeuropathies. US-guided interventions require careful coordination of transducer and needle movement along with a detailed understanding of sonoanatomy. Preprocedural planning and positioning are instrumental in performing these interventions and should not be overlooked. Minimally invasive procedures, such as corticosteroid injections, aspiration of ganglia, orthobiologics, and hydrodissection, are useful options for the nonsurgical treatment of mononeuropathies refractory to conservative care. US is effective in determining atypical anatomy such as a bifid median nerve for improved accuracy and safety during injections, and precision based techniques such as hydrodissection may aid in physically separating the nerve from scar tissue and adhesions. The most well researched US guided treatment of entrapment neuropathy is carpal tunnel syndrome which demonstrates promising results from platelet-rich plasma, prolotherapy, hydrodissection, and percutaneous needle release. However, there is significant heterogeneity in procedure technique, injectate composition, injectate volume, and periprocedural conservative care that limits interpretation of the results and warrants continued head-to-head comparisons for research. This review highlights procedural details and outcomes for US-guided interventions of the most well-studied peripheral entrapment neuropathies. Despite many promising findings for these techniques, long-term outcomes and severe nerve compression may still benefit most from surgical decompression.

Myasthenia gravis (MG) is a rare autoimmune disorder caused by autoantibodies targeting postsynaptic proteins of the neuromuscular junction. Although conventional therapies can achieve remission or sufficient symptom control in most patients with generalized MG (gMG), approximately 10%-30% of patients remain treatment refractory with an increased risk of myasthenic crises and mortality. In addition, prolonged immunosuppression can cause undesirable side effects. Over the past decade, research has focused on identifying new therapeutic targets, which paved the way to advance the development of novel therapies. Rather than exerting broad immunosuppression, novel therapies selectively target specific components involved in the immunopathogenesis of MG, such as immunoglobulin G (IgG) production (B cell-depleting agents), IgG recycling (neonatal fragment crystallizable receptor, FcRn, blocker), and complement activation (complement inhibitors). Targeted therapies have been evolving exponentially since 2017, marking a new era in treatment strategies in gMG. They have emerged as potential steroid-sparing agents with robust efficacy and favorable safety profiles. Small cohorts have demonstrated the efficacy and tolerability of these therapies during myasthenic crisis and in patients with active thymoma. Although targeted therapies have generally comparable efficacy and safety, treatment plans should be individualized, and several factors should be considered before initiating these therapies, including pharmacological spectrum (complement inhibitors are effective only in acetylcholine receptor antibody-positive MG, while FcRn and B cell-depleting agents have broader spectra), convenience of administration, and cost-effectiveness. This monograph proposes an updated treatment algorithm in refractory gMG that integrates novel targeted therapies alongside standard immunosuppressant steroid-sparing agents.

Fatigue is a burdensome symptom for pediatric populations with neuromuscular disorders, often assessed using patient- and observer-reported outcomes. The three aims of this systematic review are to: (1) explore, summarize and critically appraise primary studies that measure fatigue in pediatric neuromuscular disorders using patient- and observer-reported outcomes; (2) compare definitions of fatigue and related terms presented within these studies; and (3) summarize evidence for validation of patient- and observer-reported outcomes used in included studies. Six electronic databases were searched. Of 6801 studies identified, 64 met the inclusion criteria of measuring fatigue with a patient- and/or observer-reported outcome and investigated populations below 22 years of age with neuromuscular disorders. Fatigue assessments included questionnaires (80%), interviews/testimonials (17%), and single-item measures (3%). To measure fatigue, 50% of studies used 1 questionnaire, 28% used 2 or more questionnaires, 6% used interviews alone, 3% used single-item measures alone, and 13% used a combination of these methods. Quality appraisal indicated 80% of included studies had moderate-high risk of bias. Of all included studies, 10 employed varying definitions of fatigue or related terms. Fatigue was commonly described as an overwhelming, multidimensional and whole-body experience. Several questionnaires used to measure fatigue were developed to measure other constructs such as quality of life. Evidence for psychometric validation of all questionnaires was limited, and evidence for validation in neuromuscular disorders and pediatric populations was scarce. This review indicates a need for coherent and standardized fatigue definitions and measures while highlighting the scarcity of evidence for validation of frequently used questionnaires.