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Updates on Facioscapulohumeral Muscular Dystrophy (FSHD)
AbstractPurpose of reviewThis review aims to provide a summary of the pathophysiology, clinical presentation and management options for facioscapulohumeral dystrophy (FSHD). We discuss current management options and delve into updates about developments in targeted therapy.Recent findingsNew breakthroughs in FSHD research have led to a further understanding of aberrant DUX4 protein expression in the underlying pathophysiology of FSHD. This has paved the way for the development of targeted therapies aimed at targeting DUX4 expression or its downstream effects. Therapeutic strategies for FSHD primarily target DUX4 through th...
Source: Current Treatment Options in Neurology - April 2, 2024 Category: Neurology Source Type: research

Update on Treatment of Idiopathic (and Non-Idiopathic) Orbital Inflammation
We present the reader with a review of clinical, imaging, laboratory, and biopsy based assessment of OID and review the current treatment modalities utilized including corticosteroids, corticosteroid-sparing (immunomodulatory) agents, radiation, antibiotics, and disease specific therapy.Recent findingsTwo major developments and trends have emerged in the management of orbital inflammation. First, improved understanding and distinction of inflammation subtypes (myositis, dacryoadenitis, or infiltrative) allows for more nuanced workup and treatment. Second, immunomodulatory agents have shown promise in achieving disease cont...
Source: Current Treatment Options in Neurology - March 26, 2024 Category: Neurology Source Type: research

Treatment Approaches in Pediatric Relapsing Autoimmune Encephalitis
AbstractPurpose of reviewAutoimmune encephalitis (AE) is increasingly recognized as a treatable cause of encephalitis in children. While prior observational studies demonstrate improved motor outcomes with early immunotherapy, less is known about long-term management and treatment for relapsing disease. In this review, we present current treatment approaches to pediatric AE, in particular relapse risk and treatment for relapsing AE in children.Recent findingsA recent meta-analysis of anti-NMDAR encephalitis demonstrated that disease onset in adolescence was associated with an increased odds of relapse whereas treatment wit...
Source: Current Treatment Options in Neurology - March 21, 2024 Category: Neurology Source Type: research

Distinguishing Benign Rashes From Severe Skin Reactions From Anti-Seizure Medications
AbstractPurpose of reviewThis review describes risk factors for severe skin reactions to antiseizure medications (ASMs), the usage of updated tests to predict those with increased risk of a severe cutaneous reaction, and guides how to choose specific ASMs and dosing to lower the risk for these reactions. Information is given regarding specific mild versus severe reactions, initial diagnostic evaluation, and treatment. A table listing the risk of mild and severe cutaneous reaction risks as well as the management of potential seizures that may occur while stopping the culprit ASM are provided.Recent findingsFive new ASMs hav...
Source: Current Treatment Options in Neurology - March 21, 2024 Category: Neurology Source Type: research

Management Strategies for Atypical Parkinsonism
AbstractPurpose of ReviewAtypical parkinsonism is a term usually used to describe three neurodegenerative parkinsonian disorders: progressive supranuclear palsy (PSP), corticobasal degeneration (CBD), and multiple system atrophy (MSA). In contrast to Parkinson ’s disease, these disorders have a poor prognosis and present with a multitude of diverse symptoms, including parkinsonism, dystonia, myoclonus, gait disorders, dysarthria, dysphagia, sleep, cognitive, and behavioral disorders. In the absence of an approved disease-modifying treatment, symptomatic treatment is the mainstay of management. The purpose of this review ...
Source: Current Treatment Options in Neurology - March 21, 2024 Category: Neurology Source Type: research

Ethical Considerations in the Treatment of Cerebrovascular Disease
AbstractPurpose of ReviewTo care for patients with cerebrovascular disease (CVD), neurointerventionalists, intensivists, and other healthcare providers must be equipped to address associated ethical challenges. This review aims to delineate the applicability of fundamental bioethical approaches to CVD, highlight key ethical issues in CVD care, and delineate an ethical framework to streamline ethical decision-making for people with CVD.Recent FindingsThree introductory cases are presented. The four key principles of principalism and the approach of narrative ethics are described with reference to CVD. Key ethical considerat...
Source: Current Treatment Options in Neurology - March 15, 2024 Category: Neurology Source Type: research

Early Mobilization in Neurocritical Care
AbstractPurpose of ReviewBed rest was a treatment recommended for critically ill patients admitted to the intensive care unit (ICU) that aimed to minimize energy expenditure, permit wound healing and minimize somatic stressors. However, evidence demonstrates that bed rest leads to disuse atrophy, which may be compounded by premorbid sarcopenia and ICU-acquired weakness (ICUAW). ICUAW partly results from muscle breakdown and systemic inflammation and may exacerbate critical illness. Coupled with analgosedation, ICUAW may prolong mechanical ventilation (Kho et al. in BMJ Open Respir Res. 2019;6(1)2019; Maffei et al. in Arch ...
Source: Current Treatment Options in Neurology - February 6, 2024 Category: Neurology Source Type: research

Update on Amyloid Polyneuropathy and Treatment
AbstractPurpose of ReviewThe purpose of this review is to summarize currently available and developing diagnostic and treatment options for hereditary transthyretin amyloid polyneuropathy. Transthyretin amyloidosis (ATTR) predominantly manifests with cardiomyopathy and/or peripheral neuropathy, but amyloid deposits may be found in other organs or tissues.Recent FindingsCurrently available treatments include transthyretin gene silencers (for hereditary ATTR peripheral neuropathy only) and transthyretin stabilizers (tafamidis for ATTR cardiomyopathy in the USA, and for both hereditary ATTR peripheral neuropathy and ATTR card...
Source: Current Treatment Options in Neurology - February 1, 2024 Category: Neurology Source Type: research

Translating the ALS Genetic Revolution into Therapies: A Review
AbstractPurpose of ReviewAmyotrophic lateral sclerosis (ALS) is a neurodegenerative disease causing weakness, respiratory failure, and death within 3 to 5  years. Approximately, 10% of ALS cases have a genetic etiology (familial/fALS). The etiology of the remaining 90% of sporadic ALS (sALS) cases remains unknown. In this review, we provide an overview of approved and investigational therapies for fALS, as well as genetically informed therapeutic adv ances aimed at the larger sALS population.Recent FindingsAntisense oligonucleotides (ASOs) are a promising strategy to treat toxic gain-of-function mutations underlying most ...
Source: Current Treatment Options in Neurology - January 30, 2024 Category: Neurology Source Type: research

A New Era in the Treatment of Myasthenia Gravis: Six New Medications in The Last 6 Years
This article presents a summary of the new medications approved for generalized myasthenia gravis (gMG) since 2017. Pivotal clinical trials that led to the approval of these medications and their open-label extension studies are reviewed. We also provide information on healthcare cost when available. Lastly, we propose an approach to selecting therapies.Recent FindingsSix new medications have been approved for acetylcholine receptor antibody positive gMG. These include the complement inhibitors eculizumab, ravulizumab, and zilucoplan and the neonatal Fc receptor blockers efgartigimod, efgartigimod and hyaluronidase, and ro...
Source: Current Treatment Options in Neurology - January 23, 2024 Category: Neurology Source Type: research

Progressive Supranuclear Palsy Diagnosis and Treatment
AbstractPurpose of reviewThis review describes the current approaches to the diagnosis and management of progressive supranuclear palsy (PSP)Recent findingsPSP is an atypical parkinsonian disorder associated with the accumulation of abnormal 4-repeat tau protein in the brain. Initially, the recognized clinical phenotype included a progressive disorder with vertical supranuclear gaze palsy and prominent postural instability leading to early falls. However, the current PSP diagnostic criteria recognize a broader range of clinical PSP presentations and define eight clinical PSP variants according to the levels of diagnostic c...
Source: Current Treatment Options in Neurology - January 18, 2024 Category: Neurology Source Type: research

Vasculitic Neuropathies
AbstractPurpose of reviewTo describe the clinical presentation, diagnosis, and treatment options of vasculitic neuropathies. Overall, vasculitic neuropathies can be divided into systemic and nonsystemic. Systemic vasculitic neuropathies form part of rheumatological diseases, where in some cases, the vasculitic is the predominant feature, and these are called primary vasculitis, while in others, the vasculitis is secondary to a connective tissue disease. Nonsystemic vasculitic neuropathies involve only the peripheral nervous system, and thus, diagnosis is more challenging as it requires a high degree of suspicion and confir...
Source: Current Treatment Options in Neurology - January 11, 2024 Category: Neurology Source Type: research

The Role of Rehabilitation in Neurological Critical Care: Innovations in Early Mobilization
AbstractPurpose of reviewThis review appraises the existing literature to highlight the benefits, barriers, and role of rehabilitation in the early mobilization of patients in neurological critical care.Recent findingsWhile the benefits of early mobilization in patients with critical illness have become more widely acknowledged, there are benefits and challenges to the successful implementation of early mobilization rehabilitation programming in the neurological critical care setting. The imperative role of rehabilitation providers is underscored in successful implementation. Furthermore, the discrete needs of patients and...
Source: Current Treatment Options in Neurology - December 7, 2023 Category: Neurology Source Type: research

An Update on Palliative Care in Neurocritical Care: Providing Goal-Concordant Care in the Face of Prognostic Uncertainty
AbstractPurpose of reviewWe investigate the complexities and interplay between the concepts of prognostic uncertainty and patient preferences as they relate to the delivery of goal-concordant care to patients with severe acute brain injuries (SABI) in the Neurological Intensive Care Unit (Neuro-ICU).Recent findingsPatients with SABI in the Neuro-ICU have unique palliative care needs due to sudden, often unexpected changes in personhood and quality of life. A substantial amount of uncertainty is inherent and poses a challenge to both the patient ’s prognosis and treatment preferences. The delivery of goal-concordant care ...
Source: Current Treatment Options in Neurology - December 5, 2023 Category: Neurology Source Type: research

Emerging Principles for Treating Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD)
AbstractPurpose of reviewMyelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is a rare inflammatory disorder of the central nervous system that affects both adults and children. Neurologic disability is relapse-driven; therefore, early diagnosis and targeted treatment are critical for effective care. We review the new MOGAD diagnostic criteria and evidence for current acute and preventative therapies.Recent findingsThe International MOGAD Panel has released the first clinical, laboratory, and radiographic criteria for MOGAD diagnosis. These criteria set the stage for evaluating clinical investigations a...
Source: Current Treatment Options in Neurology - November 22, 2023 Category: Neurology Source Type: research