Advances in Treatments in Muscular Dystrophies and Motor Neuron Disorders
This article reviews advances in gene therapies, newly approved drugs, and therapeutic promises in muscular dystrophies and MNDs. (Source: Neurologic Clinics)
Source: Neurologic Clinics - November 20, 2020 Category: Neurology Authors: Bhaskar Roy, Robert Griggs Source Type: research
Update on Calcitonin Gene-Related Peptide Antagonism in the Treatment of Migraine
The discovery of calcitonin gene-related peptide (CGRP) and its role in migraine has promoted a new era in migraine treatment: CGRP antagonism. Two classes of medications are currently available: small molecules targeting the CGRP receptor and monoclonal antibodies targeting the CGRP receptor or CGRP ligand. The revolution of these medications is represented by blurring the borders between acute and preventive treatments, episodic and chronic migraine, na ïve and refractory patients and even between migraine and other headache disorders. (Source: Neurologic Clinics)
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Edoardo Caronna, Amaal J. Starling Source Type: research
Advances in the Treatment of Multiple Sclerosis
This article reviews data on available treatments along with a discussion of future treatment targets under investigation. (Source: Neurologic Clinics)
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Carolyn Goldschmidt, Marisa P. McGinley Source Type: research
Advances in the Treatment of Neuromyelitis Optica Spectrum Disorder
Neuromyelitis optica spectrum disorder (NMOSD) is a rare, relapsing-remitting neuroinflammatory disorder of the central nervous system. Advances in the understanding of NMOSD pathogenesis and identification of the NMO-specific pathogenic anti-AQP4 autoantibody have led to the development of highly effective disease-modifying strategies. Five placebo-controlled, randomized trials for NMOSD have been successfully completed as of 2020. These trials support the efficacy of rituximab and tocilizumab and led to the FDA approval of eculizumab, satralizumab and inebilizumab for NMOSD. Our review provides an update on these evidenc...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Asya Izraelit Wallach, Matthew Tremblay, Ilya Kister Source Type: research
Advances and Future Directions of Neuromodulation in Neurologic Disorders
“Deep brain stimulation is a safe and effective therapy for the management of a variety of neurologic conditions with Food and Drug Administration or humanitarian exception approval for Parkinson disease, dystonia, tremor, and obsessive-compulsive disorder. Advances in neurophysiology, neuroimagin g, and technology have driven increasing interest in the potential benefits of neurostimulation in other neuropsychiatric conditions including dementia, depression, pain, Tourette syndrome, and epilepsy, among others. New anatomic or combined targets are being investigated in these conditions to imp rove symptoms refractory...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Matthew R. Burns, Shannon Y. Chiu, Bhavana Patel, Sotiris G. Mitropanopoulos, Joshua K. Wong, Adolfo Ramirez-Zamora Source Type: research
Advances in the Management of Small Fiber Neuropathy
Small fiber neuropathy (SFN) is a prevalent neurologic syndrome. Testing methods have emerged in recent years to better diagnose it, including autonomic tests and skin punch biopsy. SFN can present in a non –length-dependent fashion and can be mistaken for syndromes such as fibromyalgia and complex regional pain syndrome. SFN is caused by a variety of metabolic, infectious, genetic, and inflammatory diseases. Recently treatments have emerged for TTR amyloid neuropathy and Fabry disease, and novel bio markers have been found both in genetic and inflammatory SFN syndromes. Ongoing trials attempt to establish the effica...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Lawrence A. Zeidman Source Type: research
Update in the Management of Myasthenia Gravis and Lambert-Eaton Myasthenic Syndrome
This article focuses on advances in management of autoimmune MG and LEMS. (Source: Neurologic Clinics)
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Cynthia Bodkin, Robert M. Pascuzzi Source Type: research
Update in the Management of Idiopathic Intracranial Hypertension
Idiopathic intracranial hypertension is a syndrome of increased intracranial pressure of unknown cause that most often occurs in women of childbearing age. Most patients are overweight or obese with a history of recent weight gain. Following a brief review of the symptoms and signs, we discuss the approach to assessment and monitoring of this condition. We then present a practical approach to treatment. Because patients frequently struggle with weight loss and can also have refractory headaches and coexisting psychosocial issues, we emphasize the importance of a multidisciplinary approach to the management of this common c...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Matthew J. Thurtell, Aki Kawasaki Source Type: research
What is New in Neuro-oncology?
Neuro-oncology is a rapidly developing field. A continuous evolution in the understanding of the molecular underpinnings of central nervous system tumors has helped reconfigure the classification of brain tumors. More importantly, it has laid the path forward for the development and investigation of new therapeutics. The authors discuss the classification of brain tumors and novel therapies in brain tumors as well as promising treatments underway. (Source: Neurologic Clinics)
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Jigisha P. Thakkar, Vikram C. Prabhu, Katherine B. Peters, Rimas V. Lukas Source Type: research
Advances in the Surgical Management of Epilepsy
This article discusses the advances in surg ical treatment of DRE in adults with focal seizures. (Source: Neurologic Clinics)
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Gregory D. Cascino, Benjamin H. Brinkmann Source Type: research
Treatment of Viral Encephalitis
Viral encephalitis is difficult to treat. Herpes simplex encephalitis has been successfully treated with acyclovir, but is still a cause for significant morbidity even with that treatment. A rare form of autoimmune encephalitis related to NMDA receptor antibody after infection by herpes simplex can be treated with corticosteroid therapy. Arthropod-borne encephalitides, such as West Nile virus encephalitis and Eastern equine encephalitis, are primarily treated with supportive measures. Attempts have been made to use immunoglobulin therapy with limited effects. Progressive multifocal leukoencephalopathy has been treated with...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Allen J. Aksamit Source Type: research
Cannabinoids in Neurologic Illnesses
Marijuana has been used to treat medical disease since well before the 1800s. Recently, increased use of cannabinoids, the chemical components of marijuana, have been seen to treat neurologic illness in children and adults. Unfortunately, data are lacking in treating most neurologic illnesses except in the field of epilepsy and pain from spasticity in multiple sclerosis. Therefore, formal conclusions about the potential efficacy, benefit, and adverse effects for most marijuana based products cannot be made at this time. Further research using gold standard scientific methodology should be performed to help address potentia...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Anup D. Patel Source Type: research
Advances and Ongoing Controversies in Patent Foramen Ovale Closure and Cryptogenic Stroke
Up to a third of strokes are cryptogenic. The prevalence of patent foramen ovale (PFO) in patients with cryptogenic stroke is higher than in individuals with stroke of known origin. It has been proposed that some cryptogenic strokes can be caused by paradoxic embolism across a PFO. The treatment of PFO includes medical treatment with antithrombotic agents and percutaneous PFO closure. There is limited evidence to support PFO closure in unselected cases of cryptogenic stroke. However, large randomized clinical trials confirmed the superiority of transcatheter PFO closure compared with medical treatment in young patients wit...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Joaquin B. Gonzalez, Fernando D. Testai Source Type: research
An Update on Botulinum Toxin in Neurology
This article gives a comprehensive overview of the clinical applications of BoNT across the field of neurology. (Source: Neurologic Clinics)
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Shannon Y. Chiu, Matthew R. Burns, Irene A. Malaty Source Type: research
Therapeutic Pitfalls in the Transition of Neurologic Patients from Pediatric to Adult Health Care Providers
Transitions of care from the pediatric to adult setting are fraught with difficulty. For patients whose neurologic problems began in childhood, there is often a lack of organized multidisciplinary care with the desired neurologic expertise in the adult setting. This monograph highlights those difficulties, reviewing disease-specific instances of the problems with transition from pediatric to adult neurologic care. The use of an arbitrary chronologic age cutoff for transition from pediatric to adult expertise in specific disease state may be a disservice in provision of care, and the disease-specific expertise of providers ...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Eugene R. Schnitzler, Michael J. Schneck Source Type: research
Therapy in Neurology
A decade ago, I had the privilege of serving as guest editor for an issue of the Neurologic Clinics dedicated to Advances in Neurologic Therapy. Who would have anticipated the proliferation of the newer, ongoing, and more selective therapeutic interventions that the current neurology practitioner has at his or her disposition? I humbly accepted serving once again as a guest editor with the specific intent of highlighting some of these state-of-the-art contemporary therapeutic advances, accepting the realization that therapeutic suggestions, although up-to-date as of this writing, mostly likely were going to be superseded b...
Source: Neurologic Clinics - November 7, 2020 Category: Neurology Authors: Jos é Biller Tags: Preface Source Type: research
This article provides a basic approach to clinical assessment and causal inference. It addresses the knowledge gap for clinical practice and provides a thematic structure to use interdisciplinary resources. (Source: Neurologic Clinics)
Source: Neurologic Clinics - October 9, 2020 Category: Neurology Authors: Mam Ibraheem, Dong Y. Han, Michael R. Dobbs Source Type: research
Toxin-Induced Acute Delirium
This article reviews the presentations of the anticholinergic toxidrome, sympathomimetic toxidrome, hallucinogenic toxidrome, γ-aminobutyric acid withdrawal, and Wernicke encephalopathy, as well as their mechanisms and basic management. (Source: Neurologic Clinics)
Source: Neurologic Clinics - October 9, 2020 Category: Neurology Authors: Alice Cai, Xuemei Cai Source Type: research
Toxin-Induced Subacute Encephalopathy
This article reviews the clinically relevant aspects of some of the more important subacute toxic encephalopathy syndromes caused by inorganic to xins, carbon monoxide, antibiotics, antineoplastic agents, and psychiatric medications. (Source: Neurologic Clinics)
Source: Neurologic Clinics - October 9, 2020 Category: Neurology Authors: David P. Lerner, Aleksey Tadevosyan, Joseph D. Burns Source Type: research
Women ’s and Fetal Issues in Neurotoxicology
This article focuses on potential toxic risk to the fetus of medications deemed necessary to manage several common maternal neurologic issues: m ultiple sclerosis, epilepsy, and headache during pregnancy and postpartum. It is important for the practitioner to have an understanding beyond the category system to understand the potential toxic risks to the infant. (Source: Neurologic Clinics)
Source: Neurologic Clinics - October 9, 2020 Category: Neurology Authors: Amy Hessler Source Type: research
Neurotoxicology Syndromes Associated with Drugs of Abuse
This article focuses on the epidemiology, pharmacology, and complications associated wit h commonly used recreational drugs, including opioids, alcohol, marijuana, cocaine, and hallucinogens. (Source: Neurologic Clinics)
Source: Neurologic Clinics - October 9, 2020 Category: Neurology Authors: Rachel A. Caplan, Jonah P. Zuflacht, Jed A. Barash, Corey R. Fehnel Source Type: research
Next Steps for Clinical Neurotoxicology
Clinical neurotoxicology is a heavy weight on those few who practice it. There are probably more than 1000 substantially neurotoxic substances, with exposure annually worldwide in the tens to hundreds of millions of people. Lead toxicity alone is reported as responsible for more than 1 million deaths yearly and devastates entire populations in cognitive development. Neurotoxic exposures may span all levels of society and the lifespan, although the developing nervous system has special risks. There are many examples of inequity in exposure risk, such as the lead exposure catastrophe of Flint, Michigan. (Source: Neurologic Clinics)
Source: Neurologic Clinics - September 13, 2020 Category: Neurology Authors: Michael R. Dobbs, Mam Ibraheem Tags: Preface Source Type: research
This article reviews key features of some of the more common or representative toxic neuropathies, including those caused by occupational and environmental exposure, medications, and chemotherapeutic agents. (Source: Neurologic Clinics)
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Michel Toledano Source Type: research
Toxin-Induced Channelopathies, Neuromuscular Junction Disorders, and Myopathy
Channelopathies, neuromuscular junction disorders, and myopathies represent multiple mechanisms by which toxins can affect the peripheral nervous system. These toxins include ciguatoxin, tetrodotoxin, botulinum toxin, metabolic poisons, venomous snake bites, and several medications. These toxins are important to be aware of because they can lead to serious symptoms, disability, or even death, and many can be treated if recognized ear. (Source: Neurologic Clinics)
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Jacqueline Janecek, Hani Kushlaf Source Type: research
Toxin-Induced Coma and Central Nervous System Depression
Many medications and toxins may induce central nervous system (CNS) depression. Even when the intention is to induce CNS depression, other nervous system adverse effects may occur, such as with anesthetics. Pain medications produce characteristic CNS toxicities. Sedative hypnotics may induce altered mentation among systemic toxicities. Stimulants may mimic coma when discontinued abruptly. Acute and chronic carbon monoxide poisoning can lead to altered mental status and prolonged cognitive difficulties. Some medications and environmental toxins can mimic brain death. High clinical suspicion and early recognition of these ef...
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Monica Krause, Sara Hocker Source Type: research
Toxin-Induced Cerebellar Disorders
The cerebellum plays an important role in motor and nonmotor systems, with damage resulting in clinical manifestations presenting as weakness, ataxia, dysarthria, and nystagmus. There are numerous environmental and industrial agents as well as medications that, through either accidental or intentional use, can result in a range of neurologic presentations. The variability in the presentation is important to recognize promptly so that early cessation in exposure, use, or abuse can be initiated to reduce the severity of symptoms. Recognition of an agent causing the particular pathology is important so that the route of expos...
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Katelyn Dolbec, Michael R. Dobbs, Mam Ibraheem Source Type: research
New toxins are emerging all the time. In this article, the authors review common toxins that cause seizure, their mechanisms, associated toxidromes, and treatments. Stimulants, cholinergic agents, gamma-aminobutyric acid antagonists, glutamate agonists, histamine and adenosine antagonists, and withdrawal states are highlighted. Understanding current mechanisms for common toxin-induced seizures can promote understanding for future toxins and predicting if seizure may occur as a result of toxicity. (Source: Neurologic Clinics)
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Haley N. Phillips, Laura Tormoehlen Source Type: research
Neurochemical and Neurobiological Weapons
Nerve agents and neurobiological weapons are among the most devastating and lethal of weapons. Acetylcholinesterase inhibitors act by increasing the amount of acetylcholine in the neuromuscular junction, resulting in flaccid paralysis. Tabun, VX, soman, and sarin are the major agents in this category. Exposure to nerve agents can be inhalational or through dermal contact. Neurotoxins may have peripheral and central effects on the nervous system. Atropine is an effective antidote to nerve agents. Neurobiological weapons entail using whole organisms or organism-synthesized toxins as agents. Some organisms that can be used as...
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: James J. Sejvar Source Type: research
Manganese Exposure and Neurologic Outcomes in Adult Populations
A review of published articles examining the effects of manganese exposure to workers and community residents shows adverse neurologic outcomes. Innovative biomarkers, including those from neuroimaging, were incorporated into many of these studies to assess both manganese exposure and neurologic outcomes. A variety of health effects were evaluated, including cognitive and motor impairments. Studies of community participants residing near manganese point sources show variability in outcomes, reflecting the complexities of exposure measurement, individual absorption, and assessment of neurologic effects. The aging population...
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Kaitlin V. Martin, David Edmondson, Kim M. Cecil, Cassandra Bezi, Miriam Leahshea Vance, Dani McBride, Erin N. Haynes Source Type: research
Neurologic Toxicities Associated with Tumor Necrosis Factor Inhibitors and Calcineurin Inhibitors
The calcineurin inhibitors cyclosporine and tacrolimus are used for their immunosuppressive effects. Neurotoxic side effects include tremor, paresthesia, and headache. Rarer neurotoxicities include seizure, posterior reversible encephalopathy syndrome, and encephalopathy. Tacrolimus tends to be more neurotoxic than cyclosporine. Management of toxicities associated with calcineurin inhibitors includes dose reduction, switching between calcineurin inhibitors, or switching to a calcineurin-free regimen. Tumor necrosis factor (TNF) inhibitors are used in autoimmune diseases. Management of demyelinating conditions among patient...
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Christopher L. Coe, Sarah N. Horst, Manhal J. Izzy Source Type: research
A Concise Review of Neurologic Complications Associated with Chimeric Antigen Receptor T-cell Immunotherapy
Chimeric antigen receptor –engineered T (CAR-T) cell immunotherapy has been successful in treating many types of hematological malignancies. CAR-T therapy, however, has been associated with toxicities, including cytokine release syndrome (CRS) as well as immune effector cell–associated neurotoxicity syndrome (ICANS). ICA NS presentation is variable, largely reversible, and manifests with encephalopathy and focal neurologic deficits. Treatment strategies largely are supportive. ICANS pathophysiology likely is related to that of CRS. Preclinical studies and clinical experience have shed light on the driving force...
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Michael W. Ruff, Elizabeth L. Siegler, Saad S. Kenderian Source Type: research
Neurotoxicity and Chemoreception
Several different types of exposure have the potential to produce olfactory and gustatory deficits related to neurotoxicity. Although the literature contains relatively few studies of such chemoreceptive dysfunction in the context of toxic exposure, this review explores the strength of such published associations. Several studies collectively demonstrated moderately strong evidence for an association between manganese dust exposure and olfactory deficits. Evidence of associations between individual chemicals, therapeutics, and composites, such as World Trade Center debris, and olfactory and gustatory deficits remains limit...
Source: Neurologic Clinics - September 11, 2020 Category: Neurology Authors: Madeline C. Aulisio, Amanda C. Glueck, Michael R. Dobbs, Sandro Pasagic, Dong Y. Han Source Type: research
This article discusses manganese, mercury, MPTP, organochlorines, organophosphates, paraquat, rotenone, and Agent Orange. (Source: Neurologic Clinics)
Source: Neurologic Clinics - September 8, 2020 Category: Neurology Authors: Steven McKnight, Nawaz Hack Source Type: research
Spinal Muscular Atrophy in the Treatment Era
Spinal muscular atrophy is an autosomal-recessive degenerative neuromuscular disease that has historically been categorized into 5 types based on the individual ’s best functional ability. Two rather remarkable treatments have recently been approved for commercial use, and both have markedly changed the natural history of this disease. Here the authors report several cases of individuals, ranging from infants to adults, to highlight diagnostic considerati ons, along with initial and long-term treatment considerations in these individuals who now have the potential for stabilization to significant improvement in funct...
Source: Neurologic Clinics - July 21, 2020 Category: Neurology Authors: Megan A. Waldrop, Bakri H. Elsheikh Source Type: research
Case Studies on the Genetic and Clinical Diagnosis of Facioscapulohumeral Muscular Dystrophy
We present 6 cases to illustrate both clinical and genetic diagnostic challenges in facioscapulohumeral muscular dystrophy and provide examples on how to navigate the different steps of genetic testing. (Source: Neurologic Clinics)
Source: Neurologic Clinics - July 21, 2020 Category: Neurology Authors: Johanna Hamel, Rabi Tawil Source Type: research
Congenital Myasthenic Syndromes
Congenital myasthenic syndromes comprise a rare heterogeneous group of diseases that impair neuromuscular transmission and are characterized by muscle fatigability and transient or permanent weakness. Symptoms are often present from birth or early childhood. These syndromes have a wide range of phenotypes and severity. Caused by genetic mutations in any of the numerous genes encoding for components of the neuromuscular junction. They are classified by where in the neuromuscular junction the mutated component is located: presynaptic, synaptic, or postsynaptic. Mutations in about 30 genes have been implicated. Diagnosis can ...
Source: Neurologic Clinics - July 21, 2020 Category: Neurology Authors: Stanley Jones P. Iyadurai Source Type: research
Limb Girdle Muscular Dystrophies
The limb girdle muscular dystrophies (LGMDs) are genetic muscle diseases with primary skeletal muscle involvement in persons with the ability to walk independently at some point in the disease course. They usually have increased creatine kinase levels along with patterns of fatty and fibrous deposition on muscle imaging and/or dystrophic features on muscle biopsy. Distinctive clinical features provide valuable diagnostic clues to the diagnosis and sometimes treatment of these disorders. The advent of gene and cell-based therapies; gene replacement, editing, and modulation; along with stem cell and small molecule therapies ...
Source: Neurologic Clinics - July 21, 2020 Category: Neurology Authors: Jacob Bockhorst, Matthew Wicklund Source Type: research
Diabetic Lumbosacral Radiculoplexus Neuropathy (Diabetic Amyotrophy)
This article provides a series of interesting cases to highlight the diagnostic challenges and discusses management decision making. (Source: Neurologic Clinics)
Source: Neurologic Clinics - July 21, 2020 Category: Neurology Authors: Melanie D. Glenn, Duaa Jabari Source Type: research
Differential Diagnoses of Inclusion Body Myositis
This article discusses atypical cases and differential diagnoses and considers the role of imaging and histopathology in differentiating inclusion body myositis. (Source: Neurologic Clinics)
Source: Neurologic Clinics - July 21, 2020 Category: Neurology Authors: Vinojini Vivekanandam, Enrico Bugiardini, Ashirwad Merve, Matthew Parton, Jasper M. Morrow, Michael G. Hanna, Pedro M. Machado Source Type: research
Small Fiber Neuropathy
Small fiber neuropathy has a broad array of presentations. Length-dependent symptoms and findings present little diagnostic difficulty, but non –length-dependent or multifocal symptoms can be challenging. Intraepidermal nerve fiber density (IENFD) testing in apparent fibromyalgia warrants further study, but skin biopsy testing of this patient population is reasonable. Avoidance of IENFD testing in situations where diagnosis of neuropathy is already clear or where neuropathy is not the cause of symptoms helps to prevent incorrect conclusions. Careful history and physical examination plus pretest probability are i...
Source: Neurologic Clinics - July 21, 2020 Category: Neurology Authors: David S. Saperstein Source Type: research