Service Lines, Neurology, and Academic Medicine: Departmental Perspectives, Implementation Strategies, and Keys to Success.

Service lines are increasingly common for organizing multidisciplinary patient care. Concerns regarding impacts of neuroscience service lines were voiced at several national neurology department chair summits, prompting the American Academy of Neurology to convene a Service Lines Workgroup. Neurology department leaders nationally at institutions that had created or considered a neuroscience service line were interviewed to elicit their experiences and lessons learned.

Respiratory Strength Training in Amyotrophic Lateral Sclerosis: A Double-Blind, Randomized, Multicenter, Sham-Controlled Trial.

Background And Objective: The objective of this study was to evaluate the short-term physiologic effect and one-year functional effect of a 12-week inspiratory and expiratory respiratory strength training (RST) program in individuals with amyotrophic lateral sclerosis (ALS).

Methods: A double-blinded, randomized, sham-controlled trial was conducted in 45 individuals with early-stage ALS. Participants were randomized into 12 weeks of active RST (30% load, n = 23) or sham RST (0% load, n = 22).

Therapies, Research Funding, and Racial Diversity in Essential Tremor: A Systematic Review of the Literature.

Background: Essential tremor (ET) is one of the most common tremor disorders in the world. Despite this, only one medication, propranolol, is approved by the Food and Drug Administration to treat it.

Objectives: We analyzed controlled clinical trials in ET, spanning the last 50 years, to identify potential shortcomings in the therapeutic clinical pipeline.

Radicava/Edaravone Findings in Biomarkers From Amyotrophic Lateral Sclerosis (REFINE-ALS): Protocol and Study Design.

Objectives: To identify putative biomarkers that may serve as quantifiable, biological, nonclinical measures of the pharmacodynamic effect of edaravone in amyotrophic lateral sclerosis (ALS) and to report real-world treatment outcomes.

Methods: This is a prospective, observational, longitudinal, multicenter (up to 40 sites) US study (Clinicaltrials.gov; NCT04259255) with at least 200 patients with ALS who will receive edaravone for 24 weeks (6 cycles; Food and Drug Administration-approved regimen).

Impact of subcutaneous immunoglobulin on quality of life in patients with chronic inflammatory demyelinating polyneuropathy previously treated with intravenous immunoglobulin.

Introduction/aims: Intravenous immunoglobulin (IVIg) is a common therapy for patients with chronic inflammatory demyelinating polyneuropathy (CIDP). IVIg may cause systemic adverse events (AEs); therefore, infusion of subcutaneous immunoglobulin (SCIg) may be preferred by some patients. In this study we document the experiences of patients transitioning from IVIg to SCIg.

Eculizumab during Pregnancy in a Patient with Treatment-Refractory Myasthenia Gravis: A Case Report.

The recombinant humanized monoclonal antibody eculizumab has been shown to be effective and well tolerated in patients with anti-acetylcholine receptor antibody-positive, treatment-refractory generalized myasthenia gravis (gMG). Myasthenia gravis (MG) often affects women of child-bearing potential. However, management can be challenging during pregnancy, and current treatment options are limited due to potential teratogenicity.

Clinical features of LRP4/agrin-antibody-positive myasthenia gravis: A multicenter study.

Introduction: Our aim in this study was to identify the prevalence and clinical characteristics of LRP4/agrin-antibody-positive double-seronegative myasthenia gravis (DNMG).

Methods: DNMG patients at 16 sites in the United States were tested for LRP4 and agrin antibodies, and the clinical data were collected.

Results: Of 181 DNMG patients, 27 (14.

An Open-Label, Prospective Study Evaluating the Clinical and Immunological Effects of Higher Dose Granulocyte Colony-Stimulating Factor in ALS.

Objective: We evaluated the safety and tolerability of higher-dose granulocyte colony-stimulating factor (G-CSF) in patients with amyotrophic lateral sclerosis. In addition, rates of disease progression and serum G-CSF levels and other immunological and hematological markers were measured.

Methods: Three patients with advanced amyotrophic lateral sclerosis were treated with G-CSF subcutaneously at 5 μg/kg twice daily for 5 consecutive days monthly for 4-12 months.

Double-blind, randomized and controlled trial of EPI-743 in Friedreich's ataxia.

Aim: To evaluate the safety and clinical effects of EPI-743 in Friedreich's ataxia patients. EPI-743 is a compound that targets oxidoreductase enzymes essential for redox control of metabolism.

Methods: We conducted a multicenter trial that evaluated EPI-743 during a 6-month placebo-controlled phase, followed by an 18-month open-label phase.

Impact of expiratory strength training in amyotrophic lateral sclerosis: Results of a randomized, sham-controlled trial.

Introduction: The purpose of this study was to determine the impact of an in-home expiratory muscle strength training (EMST) program on pulmonary, swallow, and cough function in individuals with amyotrophic lateral sclerosis (ALS).

Methods: EMST was tested in a prospective, single-center, double-blind, randomized, controlled trial of 48 ALS individuals who completed 8 weeks of either active EMST (n = 24) or sham EMST (n = 24). The primary outcome to assess treatment efficacy was change in maximum expiratory pressure (MEP).

Longitudinal gait and balance decline in Friedreich's Ataxia: A pilot study.

Introduction: Friedreich's Ataxia (FA) is a devastating, progressive, neurodegenerative disease. Objective measures that detect changes in neurological function in FA patients are needed to facilitate therapeutic clinical trials. The purpose of this pilot study was to analyze longitudinal changes in gait and balance in subjects with FA using the GAITRite Walkway System and Biodex Balance System™, respectively, and to test the ability of these measures to detect change over time compared to the Friedreich's Ataxia Rating Scale (FARS).

Muscle-Specific Receptor Tyrosine Kinase (MuSK) Myasthenia Gravis.

Autoimmune myasthenia gravis (MG) is the prototypic, antibody-mediated neuromuscular disease and is characterized by a decrease in the number of functional acetylcholine receptors (AChR) within the muscle end plate zone of the neuromuscular junction (NMJ). Although the pathophysiology of AChR-mediated myasthenia gravis has been extensively studied over the last 40 years since its original description by Patrick and Lindstrom (Science 180:871-872, 1973), less is known about the much more recently described muscle-specific kinase (MuSK) antibody-mediated MG. MuSK-MG has features clinically distinct from Ach-R MG, as well as a different pattern of response to treatment and a unique immunopathogenesis.

Voluntary Cough Airflow Differentiates Safe Versus Unsafe Swallowing in Amyotrophic Lateral Sclerosis.

Dysphagia and aspiration are prevalent in amyotrophic lateral sclerosis (ALS) and contribute to malnutrition, aspiration pneumonia, and death. Early detection of at risk individuals is critical to ensure maintenance of safe oral intake and optimal pulmonary function. We therefore aimed to determine the discriminant ability of voluntary cough airflow measures in detecting penetration/aspiration status in ALS patients.

Impact of expiratory strength training in amyotrophic lateral sclerosis.

Introduction: We evaluated the feasibility and impact of expiratory muscle strength training (EMST) on respiratory and bulbar function in persons with amyotrophic lateral sclerosis (ALS).

Methods: Twenty-five ALS patients participated in this delayed intervention open-label clinical trial. Following a lead-in period, patients completed a 5-week EMST protocol.