955 results match your criteria Neurotherapeutics [Journal]


Phosphodiesterase Inhibitors Revert Axonal Dystrophy in Friedreich's Ataxia Mouse Model.

Neurotherapeutics 2019 Feb 13. Epub 2019 Feb 13.

CIBER de Enfermedades Raras (CIBERER), Valencia, 46010, Spain.

Friedreich's ataxia (FRDA) is a neurodegenerative disorder caused by an unstable GAA repeat expansion within intron 1 of the FXN gene and characterized by peripheral neuropathy. A major feature of FRDA is frataxin deficiency with the loss of large sensory neurons of the dorsal root ganglia (DRG), namely proprioceptive neurons, undergoing dying-back neurodegeneration with progression to posterior columns of the spinal cord and cerebellar ataxia. We used isolated DRGs from a YG8R FRDA mouse model and C57BL/6J control mice for a proteomic study and a primary culture of sensory neurons from DRG to test novel pharmacological strategies. Read More

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http://dx.doi.org/10.1007/s13311-018-00706-zDOI Listing
February 2019

Efficacy of Cilostazol Administration in Alzheimer's Disease Patients with White Matter Lesions: A Positron-Emission Tomography Study.

Neurotherapeutics 2019 Feb 13. Epub 2019 Feb 13.

Department of Nuclear Medicine, Seoul National University College of Medicine & SMG-SNU Boramae Medical Center, Dongjak-gu, Seoul, Republic of Korea.

This study tested the efficacy of the phosphodiesterase type III inhibitor cilostazol in Alzheimer's disease patients with white matter lesions treated with donepezil in comparison with donepezil monotherapy using fluorodeoxyglucose (F) positron-emission tomography (FDG PET). A 24-week, randomized, double-blind, placebo-controlled, parallel-group study was conducted. Thirty-six Alzheimer's disease patients with white matter lesions who received donepezil (n = 18 each in the cilostazol and placebo groups) were enrolled. Read More

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http://dx.doi.org/10.1007/s13311-018-00708-xDOI Listing
February 2019

Differentiation Between Guillain-Barré Syndrome and Acute-Onset Chronic Inflammatory Demyelinating Polyradiculoneuritis-a Prospective Follow-up Study Using Ultrasound and Neurophysiological Measurements.

Neurotherapeutics 2019 Feb 12. Epub 2019 Feb 12.

Center of Neurology, Tübingen University Hospital, Hoppe-Seyler-Straße 3, 72076, Tübingen, Germany.

Differentiation of Guillain-Barré syndrome (GBS) and acute-onset chronic inflammatory demyelinating polyradiculoneuritis (CIDP) might be intricate in early stages. We compared electrodiagnostics (EDx) and nerve ultrasound (NUS) as tools for early distinction and follow-up. NUS and EDx have been performed at first visitation and after 6 months. Read More

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http://dx.doi.org/10.1007/s13311-019-00716-5DOI Listing
February 2019
1 Read

Nucleic Acid-Based Therapeutics for Parkinson's Disease.

Neurotherapeutics 2019 Feb 12. Epub 2019 Feb 12.

Robert Wood Johnson Medical School Institute for Neurological Therapeutics, and Department of Neurology, Rutgers Biomedical and Health Sciences, Piscataway, NJ, 08854, USA.

Parkinson's disease (PD) is a progressive neurodegenerative disorder that is diagnosed largely on clinical grounds due to characteristic motor manifestations that result from the loss of nigrostriatal dopaminergic neurons. While traditional pharmacological approaches to enhance dopamine levels, such as with L-dopa, can be very effective initially, the chronic use of this dopamine precursor is commonly plagued with motor response complications. Additionally, with advancing disease, non-motor manifestations emerge, including psychosis and dementia that compound patient disability. Read More

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http://dx.doi.org/10.1007/s13311-019-00714-7DOI Listing
February 2019

Pridopidine Induces Functional Neurorestoration Via the Sigma-1 Receptor in a Mouse Model of Parkinson's Disease.

Neurotherapeutics 2019 Feb 12. Epub 2019 Feb 12.

Basal Ganglia Pathophysiology Unit, Department of Experimental Medical Science, Lund University, BMC F11, Lund, Sweden.

Pridopidine is a small molecule in clinical development for the treatment of Huntington's disease. It was recently found to have high binding affinity to the sigma-1 receptor, a chaperone protein involved in cellular defense mechanisms and neuroplasticity. Here, we have evaluated the neuroprotective and neurorestorative effects of pridopidine in a unilateral 6-hydroxydopamine (6-OHDA) lesion model of parkinsonism in mice. Read More

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http://dx.doi.org/10.1007/s13311-018-00699-9DOI Listing
February 2019

A Longitudinal Study of the Neurologic Safety of Acute Baclofen Use After Spinal Cord Injury.

Neurotherapeutics 2019 Feb 6. Epub 2019 Feb 6.

International Collaboration on Repair Discoveries (ICORD), University of British Columbia, 818 West 10th Avenue, Vancouver, British Columbia, V5Z 1M9, Canada.

The objective of our study was to determine whether treatment with baclofen is neurologically safe with respect to exposure during recovery from spinal cord injury. We performed a secondary longitudinal analysis of a cohort of adult patients with traumatic acute spinal cord injury. Cumulative baclofen dose was computed over the first 4 weeks following injury from concomitant medication information from a completed clinical trial. Read More

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http://dx.doi.org/10.1007/s13311-019-00713-8DOI Listing
February 2019
2 Reads

Anti-Inflammatory and Neuroprotective Effects of DIPOPA (N,N-Diisopropyl-2-Oxopropanamide), an Ethyl Pyruvate Bioisoster, in the Postischemic Brain.

Neurotherapeutics 2019 Jan 24. Epub 2019 Jan 24.

Department of Anatomy, Inha University School of Medicine, Michuhol-gu Inharo 100, Inchon, 22202, Republic of Korea.

Ethyl pyruvate (EP) is a simple aliphatic ester of pyruvic acid and has been shown to have protective properties, which have been attributed to its anti-inflammatory, anti-oxidative, and anti-apoptotic functions. In an effort to develop better derivatives of EP, we previously synthesized DEOPA (N,N-diethyl-2-oxopropanamide, a novel isoster of EP) which has greater neuroprotective effects than EP, probably due to its anti-inflammatory and anti-excitotoxic effects. In the present study, we synthesized 3 DEOPA derivatives, in which its diethylamino group was substituted with diisopropylamino, dipropylamino, or diisobutylamino groups. Read More

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http://dx.doi.org/10.1007/s13311-019-00711-wDOI Listing
January 2019

Stereoelectroencephalography Versus Subdural Electrodes for Localization of the Epileptogenic Zone: What Is the Evidence?

Neurotherapeutics 2019 Jan;16(1):59-66

Department of Neurological Surgery, University of Pittsburgh, Pittsburgh, PA, 15238, USA.

Accurate and safe localization of epileptic foci is the crux of surgical therapy for focal epilepsy. As an initial evaluation, patients with drug-resistant epilepsy often undergo evaluation by noninvasive methods to identify the epileptic focus (i.e. Read More

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http://dx.doi.org/10.1007/s13311-018-00703-2DOI Listing
January 2019

A New Era for Surgical Neurotherapeutics.

Neurotherapeutics 2019 Jan;16(1):1-2

Center for the Neural Basis of Cognition, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.

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http://dx.doi.org/10.1007/s13311-019-00709-4DOI Listing
January 2019

Oligonucleotide Therapeutics as a New Class of Drugs for Malignant Brain Tumors: Targeting mRNAs, Regulatory RNAs, Mutations, Combinations, and Beyond.

Neurotherapeutics 2019 Jan 14. Epub 2019 Jan 14.

Ann Romney Center for Neurologic Diseases, Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Initiative for RNA Medicine, Boston, Massachusetts, 02115, USA.

Malignant brain tumors are rapidly progressive and often fatal owing to resistance to therapies and based on their complex biology, heterogeneity, and isolation from systemic circulation. Glioblastoma is the most common and most aggressive primary brain tumor, has high mortality, and affects both children and adults. Despite significant advances in understanding the pathology, multiple clinical trials employing various treatment strategies have failed. Read More

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http://dx.doi.org/10.1007/s13311-018-00702-3DOI Listing
January 2019

Herpes Viruses, Alzheimer's Disease, and Related Dementias: Unifying or Confusing Hypothesis?

Authors:
Avindra Nath

Neurotherapeutics 2019 Jan;16(1):180-181

Section of Infections of the Nervous System, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bldg 10; Room 7C-103, 10 Center Drive, Bethesda, MD, 20892, USA.

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http://dx.doi.org/10.1007/s13311-018-00701-4DOI Listing
January 2019

Nucleic Acid Therapies for Ischemic Stroke.

Neurotherapeutics 2019 Jan 11. Epub 2019 Jan 11.

Division of Neurocritical Care, Department of Anesthesiology and Critical Care Medicine, Johns Hopkins University, 600 N. Wolfe Street, Baltimore, MD, 21287, USA.

Stroke remains a leading cause of disability and death worldwide despite significant scientific and therapeutic advances. Therefore, there is a critical need to improve stroke prevention and treatment. In this review, we describe several examples that leverage nucleic acid therapeutics to improve stroke care through prevention, acute treatment, and recovery. Read More

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http://dx.doi.org/10.1007/s13311-019-00710-xDOI Listing
January 2019
2 Reads

Plasma Periostin and Delayed Cerebral Ischemia After Aneurysmal Subarachnoid Hemorrhage.

Neurotherapeutics 2019 Jan 11. Epub 2019 Jan 11.

Department of Neurosurgery, Mie University Graduate School of Medicine, 2-174 Edobashi, Tsu, Mie, 514-8507, Japan.

Delayed cerebral ischemia (DCI) is a serious complication of aneurysmal subarachnoid hemorrhage (SAH). Matricellular protein periostin (POSTN) has been found to be upregulated and linked with early brain injury after experimental SAH. The aim of the present study was to investigate the relationship between plasma POSTN levels and various clinical factors including serum levels of C-reactive protein (CRP), an inflammatory marker, in 109 consecutive SAH patients whose POSTN levels were measured at days 1-12 after aneurysmal obliteration. Read More

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http://dx.doi.org/10.1007/s13311-018-00707-yDOI Listing
January 2019
1 Read

Prostaglandin A1 Inhibits the Cognitive Decline of APP/PS1 Transgenic Mice via PPARγ/ABCA1-dependent Cholesterol Efflux Mechanisms.

Neurotherapeutics 2019 Jan 9. Epub 2019 Jan 9.

College of Life and Health Sciences, Northeastern University, No. 3-11. Wenhua Road, Shenyang, 110819, People's Republic of China.

Prostaglandins (PGs) are early and key contributors to chronic neurodegenerative diseases. As one important member of classical PGs, PGA1 has been reported to exert potential neuroprotective effects. However, the mechanisms remain unknown. Read More

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http://dx.doi.org/10.1007/s13311-018-00704-1DOI Listing
January 2019
1 Read

Herpes Infections and Dementia: Rebutting Alternative Fact.

Neurotherapeutics 2019 Jan;16(1):176-179

Nuffield Department of Clinical Neurosciences, John Radcliffe Hospital, Oxford, UK.

Recent commentary in Neurotherapeutics by Nath critically addresses the earlier report by Tzeng et al. that aggressive antiviral treatment (AVT) against herpes simplex virus (HSV) was associated with a later decrease in the incidence of Alzheimer's disease (AD). Nath raises issues that we respond to: we point out that (i) the treated group (probably with severe infection) is likely to harbor genetic risk alleles that predispose to both AD and HSV infection-the potential treatment bias cited by Nath would support (rather than challenge) the preventive effect of AVT; (ii) HSV is well known to establish persistent infection in the brain; and (iii) current AVT compounds used to combat herpes viruses are highly specific for this class of viruses. Read More

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http://link.springer.com/10.1007/s13311-018-00700-5
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http://dx.doi.org/10.1007/s13311-018-00700-5DOI Listing
January 2019
6 Reads

The Potential for a Speech Brain-Computer Interface Using Chronic Electrocorticography.

Neurotherapeutics 2019 Jan;16(1):144-165

Department of Neurology, The Johns Hopkins University School of Medicine, Baltimore, MD, USA.

A brain-computer interface (BCI) is a technology that uses neural features to restore or augment the capabilities of its user. A BCI for speech would enable communication in real time via neural correlates of attempted or imagined speech. Such a technology would potentially restore communication and improve quality of life for locked-in patients and other patients with severe communication disorders. Read More

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http://link.springer.com/10.1007/s13311-018-00692-2
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http://dx.doi.org/10.1007/s13311-018-00692-2DOI Listing
January 2019
6 Reads

Toward Electrophysiology-Based Intelligent Adaptive Deep Brain Stimulation for Movement Disorders.

Neurotherapeutics 2019 Jan;16(1):105-118

Department of Neurological Surgery, University of Pittsburgh, Pittsburgh, PA, USA.

Deep brain stimulation (DBS) represents one of the major clinical breakthroughs in the age of translational neuroscience. In 1987, Benabid and colleagues demonstrated that high-frequency stimulation can mimic the effects of ablative neurosurgery in Parkinson's disease (PD), while offering two key advantages to previous procedures: adjustability and reversibility. Deep brain stimulation is now an established therapeutic approach that robustly alleviates symptoms in patients with movement disorders, such as Parkinson's disease, essential tremor, and dystonia, who present with inadequate or adverse responses to medication. Read More

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http://dx.doi.org/10.1007/s13311-018-00705-0DOI Listing
January 2019
1 Read

Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias.

Neurotherapeutics 2019 Jan 3. Epub 2019 Jan 3.

Department of Human Genetics, LUMC, P.O. Box 9600, 2300 RC, Leiden, The Netherlands.

Autosomal dominant cerebellar ataxias (ADCAs) are a group of neurodegenerative disorders characterized by degeneration of the cerebellum and its connections. All ADCAs have progressive ataxia as their main clinical feature, frequently accompanied by dysarthria and oculomotor deficits. The most common spinocerebellar ataxias (SCAs) are 6 polyglutamine (polyQ) SCAs. Read More

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http://link.springer.com/10.1007/s13311-018-00696-y
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http://dx.doi.org/10.1007/s13311-018-00696-yDOI Listing
January 2019
2 Reads

Spatial Training Ameliorates Long-Term Alzheimer's Disease-Like Pathological Deficits by Reducing NLRP3 Inflammasomes in PR5 Mice.

Neurotherapeutics 2018 Dec 17. Epub 2018 Dec 17.

Department of Neuropsychiatry, Affiliated Zhongda Hospital, School of Medicine, Southeast University, Nanjing, China.

Recent studies have suggested that cognitive training could delay memory loss in Alzheimer's disease (AD). However, whether and how cognitive training produces long-term benefits remains unclear. Here, 10-month-old PR5 mice were spatially trained in a water maze for 4 consecutive weeks. Read More

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http://dx.doi.org/10.1007/s13311-018-00698-wDOI Listing
December 2018

Gene Therapy for Neurodegenerative Diseases.

Neurotherapeutics 2019 Jan;16(1):166-175

Brain Modulation Laboratory, Department of Neurological Surgery, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, 15213, USA.

Gene therapy has the potential to provide therapeutic benefit to millions of people with neurodegenerative diseases through several means, including direct correction of pathogenic mechanisms, neuroprotection, neurorestoration, and symptom control. Therapeutic efficacy is therefore dependent on knowledge of the disease pathogenesis and the required temporal and spatial specificity of gene expression. An additional critical challenge is achieving the most complete transduction of the target structure while avoiding leakage into neighboring regions or perivascular spaces. Read More

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http://dx.doi.org/10.1007/s13311-018-00694-0DOI Listing
January 2019

The Role of the Peripheral Nerve Surgeon in the Treatment of Pain.

Neurotherapeutics 2019 Jan;16(1):9-25

Division of Plastic and Reconstructive Surgery, Washington University in St. Louis, St. Louis, MO, USA.

Pain is a frequent cause of physician visits. Many physicians find these patients challenging because they often have complicated histories, emotional comorbidities, confusing examinations, difficult problems to fix, and the possibility of factitious complaints for attention or narcotic pain medications. As a result, many patients are lumped into the category of chronic, centralized pain and relegated to pain management. Read More

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http://dx.doi.org/10.1007/s13311-018-00695-zDOI Listing
January 2019

Tractography for Surgical Neuro-Oncology Planning: Towards a Gold Standard.

Neurotherapeutics 2019 Jan;16(1):36-51

Department of Neurosurgery, Stanford University, 300 Pasteur Drive, Palo Alto, CA, 94304, USA.

Magnetic resonance imaging tractography permits in vivo visualization of white matter structures. Aside from its academic value, tractography has been proven particularly useful to neurosurgeons for preoperative planning. Preoperative tractography permits both qualitative and quantitative analyses of tumor effects upon surrounding white matter, allowing the surgeon to specifically tailor their operative approach. Read More

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http://dx.doi.org/10.1007/s13311-018-00697-xDOI Listing
January 2019

Optimizing Trajectories for Cranial Laser Interstitial Thermal Therapy Using Computer-Assisted Planning: A Machine Learning Approach.

Neurotherapeutics 2019 Jan;16(1):182-191

Department of Clinical and Experimental Epilepsy, UCL Institute of Neurology, 33 Queen Square, London, WC1E 6BT, UK.

Laser interstitial thermal therapy (LITT) is an alternative to open surgery for drug-resistant focal mesial temporal lobe epilepsy (MTLE). Studies suggest maximal ablation of the mesial hippocampal head and amygdalohippocampal complex (AHC) improves seizure freedom rates while better neuropsychological outcomes are associated with sparing of the parahippocampal gyrus (PHG). Optimal trajectories avoid sulci and CSF cavities and maximize distance from vasculature. Read More

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http://link.springer.com/10.1007/s13311-018-00693-1
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http://dx.doi.org/10.1007/s13311-018-00693-1DOI Listing
January 2019
11 Reads

TET1 Overexpression Mitigates Neuropathic Pain Through Rescuing the Expression of μ-Opioid Receptor and Kv1.2 in the Primary Sensory Neurons.

Neurotherapeutics 2018 Dec 4. Epub 2018 Dec 4.

Department of Anesthesiology, New Jersey Medical School, Rutgers, The State University of New Jersey, 185 S. Orange Ave., MSB, E-661, Newark, NJ, 07103, USA.

Peripheral nerve injury downregulates the expression of the μ-opioid receptor (MOR) and voltage-gated potassium channel subunit Kv1.2 by increasing their DNA methylation in the dorsal root ganglion (DRG). Ten-eleven translocation methylcytosine dioxygenase 1 (TET1) causes DNA demethylation. Read More

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http://dx.doi.org/10.1007/s13311-018-00689-xDOI Listing
December 2018

Focused Ultrasound for Neuromodulation.

Authors:
David P Darrow

Neurotherapeutics 2019 Jan;16(1):88-99

Department of Neurosurgery, University of Minnesota, 420 Delaware St SE, MMC 96, Room D-429, Minneapolis, MN, 55455, USA.

For more than 70 years, the promise of noninvasive neuromodulation using focused ultrasound has been growing while diagnostic ultrasound established itself as a foundation of clinical imaging. Significant technical challenges have been overcome to allow transcranial focused ultrasound to deliver spatially restricted energy into the nervous system at a wide range of intensities. High-intensity focused ultrasound produces reliable permanent lesions within the brain, and low-intensity focused ultrasound has been reported to both excite and inhibit neural activity reversibly. Read More

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http://dx.doi.org/10.1007/s13311-018-00691-3DOI Listing
January 2019
7 Reads

Behavioral and Cognitive Improvement Induced by Novel Imidazoline I Receptor Ligands in Female SAMP8 Mice.

Neurotherapeutics 2018 Nov 20. Epub 2018 Nov 20.

Pharmacology Section, Department of Pharmacology, Toxicology and Medicinal Chemistry, Faculty of Pharmacy and Food Sciences, and Institut de Neurociències, University of Barcelona, Av. Joan XXIII, 27-31, 08028, Barcelona, Spain.

As populations increase their life expectancy, age-related neurodegenerative disorders such as Alzheimer's disease have become more common. I-Imidazoline receptors (I-IR) are widely distributed in the central nervous system, and dysregulation of I-IR in patients with neurodegenerative diseases has been reported, suggesting their implication in cognitive impairment. This evidence indicates that high-affinity selective I-IR ligands potentially contribute to the delay of neurodegeneration. Read More

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http://link.springer.com/10.1007/s13311-018-00681-5
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http://dx.doi.org/10.1007/s13311-018-00681-5DOI Listing
November 2018
9 Reads

The Evolution of Selective Dorsal Rhizotomy for the Management of Spasticity.

Neurotherapeutics 2019 Jan;16(1):3-8

Division of Neurosurgery, Grootte Schuur Hospital, OMB H53, University of Cape Town, Observatory, Cape Town, South Africa.

Selective dorsal rhizotomy is a key technique in the surgical management of spasticity in patients with cerebral palsy. The technique evolved from the late 1800s when pioneers like Dana and Abbe performed dorsal rhizotomy in their treatment of refractory pain. These surgeons noted a reduction in muscle tone associated with the operation. Read More

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http://dx.doi.org/10.1007/s13311-018-00690-4DOI Listing
January 2019
13 Reads

Myopathy: Recent Progress, Current Therapies, and Future Directions.

Neurotherapeutics 2018 10;15(4):837-839

Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.

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http://link.springer.com/10.1007/s13311-018-00688-y
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http://dx.doi.org/10.1007/s13311-018-00688-yDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277290PMC
October 2018
14 Reads

Dynamin 2 (DNM2) as Cause of, and Modifier for, Human Neuromuscular Disease.

Neurotherapeutics 2018 10;15(4):966-975

Genetics and Genome Biology Program, Hospital for Sick Children, Toronto, ON, M5G 0A4, Canada.

Dynamin 2 (DNM2) belongs to a family of large GTPases that are well known for mediating membrane fission by oligomerizing at the neck of membrane invaginations. Autosomal dominant mutations in the ubiquitously expressed DNM2 cause 2 discrete neuromuscular diseases: autosomal dominant centronuclear myopathy (ADCNM) and dominant intermediate Charcot-Marie-Tooth neuropathy (CMT). CNM and CMT mutations may affect DNM2 in distinct manners: CNM mutations may cause protein hyperactivity with elevated GTPase and fission activities, while CMT mutations could impair DNM2 lipid binding and activity. Read More

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http://link.springer.com/10.1007/s13311-018-00686-0
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http://dx.doi.org/10.1007/s13311-018-00686-0DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277281PMC
October 2018
8 Reads

Correction to: The Therapy of Congenital Myasthenic Syndromes.

Authors:
Andrew G Engel

Neurotherapeutics 2019 Jan;16(1):244

Department of Neurology, Mayo Clinic, Rochester, MN, USA.

The third paragraph in the left column of page 256 of the article pertaining to the treatment of congenital choline acetyl transferase (ChAT) deficiency states that "Because apneic attacks occur suddenly in infants and children, the parents should be provided with an inflatable rescue bag." Read More

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http://dx.doi.org/10.1007/s13311-018-00672-6DOI Listing
January 2019
1 Read

Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy.

Authors:
Perry B Shieh

Neurotherapeutics 2018 10;15(4):840-848

Department of Neurology, University of California, Los Angeles, 300 Medical Plaza, Suite B-200, Los Angeles, CA, 90095, USA.

Duchenne muscular dystrophy (DMD) is a progressive X-linked degenerative muscle disease due to mutations in the DMD gene. Genetic confirmation has become standard in recent years. Improvements in the standard of care for DMD have led to improved survival. Read More

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http://link.springer.com/10.1007/s13311-018-00687-z
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http://dx.doi.org/10.1007/s13311-018-00687-zDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277306PMC
October 2018
13 Reads

Ryanodine Receptor 1-Related Myopathies: Diagnostic and Therapeutic Approaches.

Neurotherapeutics 2018 10;15(4):885-899

Neuromuscular Symptoms Unit, National Institute of Nursing Research, National Institutes of Health, Bethesda, MD, USA.

Ryanodine receptor type 1-related myopathies (RYR1-RM) are the most common class of congenital myopathies. Historically, RYR1-RM classification and diagnosis have been guided by histopathologic findings on muscle biopsy. Main histological subtypes of RYR1-RM include central core disease, multiminicore disease, core-rod myopathy, centronuclear myopathy, and congenital fiber-type disproportion. Read More

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http://link.springer.com/10.1007/s13311-018-00677-1
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http://dx.doi.org/10.1007/s13311-018-00677-1DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277304PMC
October 2018
5 Reads

Diagnosis and Treatment of Mitochondrial Myopathies.

Neurotherapeutics 2018 10;15(4):943-953

Wellcome Centre for Mitochondrial Research, Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, UK.

Mitochondrial myopathies are progressive muscle conditions caused primarily by the impairment of oxidative phosphorylation (OXPHOS) in the mitochondria. This causes a deficit in energy production in the form of adenosine triphosphate (ATP), particularly in skeletal muscle. The diagnosis of mitochondrial myopathy is reliant on the combination of numerous techniques including traditional histochemical, immunohistochemical, and biochemical testing combined with the fast-emerging molecular genetic techniques, namely next-generation sequencing (NGS). Read More

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http://dx.doi.org/10.1007/s13311-018-00674-4DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277287PMC
October 2018
1 Read

Applications of Focused Ultrasound in Cerebrovascular Diseases and Brain Tumors.

Neurotherapeutics 2019 Jan;16(1):67-87

Focused Ultrasound Foundation, Charlottesville, Virginia, USA.

Oncology and cerebrovascular disease constitute two of the most common diseases afflicting the central nervous system. Standard of treatment of these pathologies is based on multidisciplinary approaches encompassing combination of interventional procedures such as open and endovascular surgeries, drugs (chemotherapies, anti-coagulants, anti-platelet therapies, thrombolytics), and radiation therapies. In this context, therapeutic ultrasound could represent a novel diagnostic/therapeutic in the armamentarium of the surgeon to treat these diseases. Read More

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http://link.springer.com/10.1007/s13311-018-00683-3
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http://dx.doi.org/10.1007/s13311-018-00683-3DOI Listing
January 2019
24 Reads

Myopathies Related to Glycogen Metabolism Disorders.

Neurotherapeutics 2018 10;15(4):915-927

Division of Neuromuscular & Neurometabolic Disorders, Departments of Pediatrics and Medicine, McMaster University, Hamilton Health Sciences Centre, Rm 2H26, Hamilton, ON, L8S 4L8, Canada.

Most of the glycogen metabolism disorders that affect skeletal muscle involve enzymes in glycogenolysis (myophosphorylase (PYGM), glycogen debranching enzyme (AGL), phosphorylase b kinase (PHKB)) and glycolysis (phosphofructokinase (PFK), phosphoglycerate mutase (PGAM2), aldolase A (ALDOA), β-enolase (ENO3)); however, 3 involve glycogen synthesis (glycogenin-1 (GYG1), glycogen synthase (GSE), and branching enzyme (GBE1)). Many present with exercise-induced cramps and rhabdomyolysis with higher-intensity exercise (i.e. Read More

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http://link.springer.com/10.1007/s13311-018-00684-2
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http://dx.doi.org/10.1007/s13311-018-00684-2DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277299PMC
October 2018
17 Reads

Closed-Loop Brain Stimulation for Drug-Resistant Epilepsy: Towards an Evidence-Based Approach to Personalized Medicine.

Neurotherapeutics 2019 Jan;16(1):119-127

Brain Modulation Lab, Department of Neurological Surgery, University of Pittsburgh, Pittsburgh, PA, USA.

Closed-loop brain stimulation is one of the few treatments available for patients who are ineligible for traditional surgical resection of the epileptogenic zone, due to having generalized epilepsy, multifocal epilepsy, or focal epilepsy localized to an eloquent brain region. Due to its clinical efficacy and potential to delivery personalized therapy based on an individual's own intracerebral electrophysiology, this treatment is becoming an important part of clinical practice, despite a limited understanding of how to program detection and stimulation parameters for optimal, patient-specific benefit. To bring this challenge into focus, we review the evolution of neural stimulation for epilepsy, provide a technical overview of the RNS System (the only FDA-approved closed-loop device), and discuss the major challenges of working with a closed-loop device. Read More

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http://dx.doi.org/10.1007/s13311-018-00682-4DOI Listing
January 2019

Invasive Neuromodulation for the Treatment of Pediatric Epilepsy.

Neurotherapeutics 2019 Jan;16(1):128-133

Division of Neurosurgery, Hospital for Sick Children, Department of Surgery, University of Toronto, 1503 555 University Ave., Toronto, ON, M5G 1X8, Canada.

Neuromodulatory strategies are increasingly adopted for the treatment of intractable epilepsy in children. These encompass a wide range of treatments aimed at externally stimulating neural circuitry in order to decrease seizure frequency. In the current review, the authors discuss the evidence for invasive neuromodulation, namely vagus nerve and deep brain stimulation in affected children. Read More

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http://dx.doi.org/10.1007/s13311-018-00685-1DOI Listing
January 2019
1 Read

Venlafaxine Mitigates Depressive-Like Behavior in Ovariectomized Rats by Activating the EPO/EPOR/JAK2 Signaling Pathway and Increasing the Serum Estradiol Level.

Neurotherapeutics 2018 Oct 25. Epub 2018 Oct 25.

Department of Biochemistry, Faculty of Pharmacy, Cairo University, Cairo, Egypt.

Reduced estradiol levels are associated with depression in women during the transition to and after menopause. A considerable number of studies focusing on the theme of treating depression through the activation of erythropoietin (EPO)-induced signaling pathways have been published. Venlafaxine is an approved antidepressant drug that inhibits both serotonin and norepinephrine transporters. Read More

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http://link.springer.com/10.1007/s13311-018-00680-6
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http://dx.doi.org/10.1007/s13311-018-00680-6DOI Listing
October 2018
12 Reads

Facioscapulohumeral Muscular Dystrophy: Update on Pathogenesis and Future Treatments.

Neurotherapeutics 2018 10;15(4):863-871

Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673, Rochester, NY, 14642, USA.

A reliable model of a disease pathomechanism is the first step to develop targeted treatment. In facioscapulohumeral muscular dystrophy (FSHD), the third most common muscular dystrophy, recent advances in understanding the complex genetics and epigenetics have led to the identification of a disease mechanism, moving the field towards targeted therapy development. FSHD is caused by expression of DUX4, a retrogene located on the D4Z4 macrosatellite repeat array on chromosome 4q35, a gene expressed in the germline but typically repressed in somatic tissue. Read More

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http://dx.doi.org/10.1007/s13311-018-00675-3DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277282PMC
October 2018

Skeletal Muscle Channelopathies.

Neurotherapeutics 2018 10;15(4):954-965

Department of Neurology and Neurotherapeutics, UT Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, Texas, 75390, USA.

Skeletal muscle channelopathies are rare heterogeneous diseases with marked genotypic and phenotypic variability. These disorders cause lifetime disability and impact quality of life. Despite advances in understanding of the molecular pathology of these disorders, the diverse phenotypic manifestations remain a challenge in diagnosis, therapeutic, genetic counseling, and research planning. Read More

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http://dx.doi.org/10.1007/s13311-018-00678-0DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277285PMC
October 2018

Autoimmune Myopathies: Updates on Evaluation and Treatment.

Neurotherapeutics 2018 10;15(4):976-994

Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, 60 Fenwood Road, Boston, MA, 02115, USA.

The major forms of autoimmune myopathies include dermatomyositis (DM), polymyositis (PM), myositis associated with antisynthetase syndrome (ASS), immune-mediated necrotizing myopathy (IMNM), and inclusion body myositis (IBM). While each of these conditions has unique clinical and histopathological features, they all share an immune-mediated component. These conditions can occur in isolation or can be associated with systemic malignancies or connective tissue disorders (overlap syndromes). Read More

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http://dx.doi.org/10.1007/s13311-018-00676-2DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277300PMC
October 2018
2 Reads

Myotonic Dystrophies: Targeting Therapies for Multisystem Disease.

Neurotherapeutics 2018 10;15(4):872-884

Department of Neurology, The Ohio State University, 395 West 12th Avenue, Columbus, OH, 43210, USA.

Myotonic dystrophy is an autosomal dominant muscular dystrophy not only associated with muscle weakness, atrophy, and myotonia but also prominent multisystem involvement. There are 2 similar, but distinct, forms of myotonic dystrophy; type 1 is caused by a CTG repeat expansion in the DMPK gene, and type 2 is caused by a CCTG repeat expansion in the CNBP gene. Type 1 is associated with distal limb, neck flexor, and bulbar weakness and results in different phenotypic subtypes with variable onset from congenital to very late-onset as well as variable signs and symptoms. Read More

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http://link.springer.com/10.1007/s13311-018-00679-z
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http://dx.doi.org/10.1007/s13311-018-00679-zDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277298PMC
October 2018
20 Reads

GNE Myopathy: Etiology, Diagnosis, and Therapeutic Challenges.

Neurotherapeutics 2018 10;15(4):900-914

Medical Genetics Branch, National Human Genome Research Institute (NHGRI), National Institutes of Health, Bethesda, MD, 20892, USA.

GNE myopathy, previously known as hereditary inclusion body myopathy (HIBM), or Nonaka myopathy, is a rare autosomal recessive muscle disease characterized by progressive skeletal muscle atrophy. It has an estimated prevalence of 1 to 9:1,000,000. GNE myopathy is caused by mutations in the GNE gene which encodes the rate-limiting enzyme of sialic acid biosynthesis. Read More

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http://dx.doi.org/10.1007/s13311-018-0671-yDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277305PMC
October 2018
1 Read

L-Norvaline Reverses Cognitive Decline and Synaptic Loss in a Murine Model of Alzheimer's Disease.

Neurotherapeutics 2018 10;15(4):1036-1054

Drug Discovery Laboratory, The Azrieli Faculty of Medicine, Bar-Ilan University, 1311502, Safed, Israel.

The urea cycle is strongly implicated in the pathogenesis of Alzheimer's disease (AD). Arginase-I (ARGI) accumulation at sites of amyloid-beta (Aβ) deposition is associated with L-arginine deprivation and neurodegeneration. An interaction between the arginase II (ARGII) and mTOR-ribosomal protein S6 kinase β-1 (S6K1) pathways promotes inflammation and oxidative stress. Read More

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http://dx.doi.org/10.1007/s13311-018-0669-5DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277292PMC
October 2018

Novel Uses of Nerve Transfers.

Authors:
Thomas J Wilson

Neurotherapeutics 2019 Jan;16(1):26-35

Department of Neurosurgery, Stanford University, 300 Pasteur Drive, R293, Stanford, California, 94305, USA.

Nerve transfer surgery involves using a working, functional nerve with an expendable or duplicated function as a donor to supply axons and restore function to an injured recipient nerve. Nerve transfers were originally popularized for the restoration of motor function in patients with peripheral nerve injuries. However, more recently, novel uses of nerve transfers have been described, including nerve transfers for sensory reinnervation, nerve transfers for spinal cord injury and stroke patients, supercharge end-to-side nerve transfers, and targeted muscle reinnervation for the prevention and treatment of postamputation neuroma pain. Read More

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http://link.springer.com/10.1007/s13311-018-0664-x
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http://dx.doi.org/10.1007/s13311-018-0664-xDOI Listing
January 2019
12 Reads

Statin-Associated Muscle Disease: Advances in Diagnosis and Management.

Neurotherapeutics 2018 10;15(4):1006-1017

Division of Cardiology, Hartford Healthcare, Hartford, CT, USA.

Since the first approval of lovastatin in 1987, hydroxy-methyl-glutaryl CoA (HMG CoA) reductase inhibitors, or statins, have been effective and widely popular cholesterol-lowering agents with substantial benefits for the prevention and treatment of cardiovascular disease. Not all patients can tolerate these drugs, however, and statin intolerance is most frequently associated with a range of side effects directed toward skeletal muscle, termed statin-associated muscle symptoms or SAMS. SAMS are particularly difficult to treat because there are no validated biomarkers or tests that can be used to confirm patient self-reports of SAMS, and a number of patients who report SAMS have non-specific muscle pain not attributable to statin therapy. Read More

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http://dx.doi.org/10.1007/s13311-018-0670-zDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277297PMC
October 2018
1 Read

Can Arginase Inhibitors Be the Answer to Therapeutic Challenges in Alzheimer's Disease?

Neurotherapeutics 2018 10;15(4):1032-1035

International Centre for Neurotherapeutics, Dublin City University, 9, Dublin, Ireland.

While the extensive hunt for therapeutics combating Alzheimer's disease (AD) has fallen short of delivering effective treatments, breakthroughs towards understanding the disease mechanisms and identifying areas for future research have nevertheless been enabled. The majority of clinical trials with β- and γ-secretase modulators have been suspended from additional studies or terminated due to toxicity issues and health concerns. The lack of progress in developing innovative AD therapies has also prompted a resurgence of interest in more traditional symptomatic treatments with cholinesterase inhibitors and N-methyl-D-aspartate receptor antagonists, as well as in the research of immune response modulators. Read More

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http://dx.doi.org/10.1007/s13311-018-0668-6DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277284PMC
October 2018
1 Read

Directional Deep Brain Stimulation.

Neurotherapeutics 2019 Jan;16(1):100-104

Department of Neurology, University Hospital Würzburg, Josef-Schneider-Str. 11, D-97080, Würzburg, Germany.

Over the last years, deep brain stimulation has seen many technological innovations. New electrode designs allowing to direct the current flow not only in the vertical but also in the horizontal plane are the most recent. We summarize the concept of "directional deep brain stimulation" with its opportunities and challenges and the available study data and discuss the use of imaging techniques to assist programming deep brain stimulation devices. Read More

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http://dx.doi.org/10.1007/s13311-018-0667-7DOI Listing
January 2019
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Evaluation of 3K3A-Activated Protein C to Treat Neonatal Hypoxic Ischemic Brain Injury in the Spiny Mouse.

Neurotherapeutics 2019 Jan;16(1):231-243

The Ritchie Centre, Hudson Institute of Medical Research, 27-31 Wright St, Clayton, Melbourne, 3168, Australia.

Neonatal hypoxic ischemic encephalopathy (HIE) resulting from intrapartum asphyxia is a global problem that causes severe disabilities and up to 1 million deaths annually. A variant form of activated protein C, 3K3A-APC, has cytoprotective properties that attenuate brain injury in models of adult stroke. In this study, we compared the ability of 3K3A-APC and APC (wild-type (wt)) to attenuate neonatal brain injury, using the spiny mouse (Acomys cahirinus) model of intrapartum asphyxia. Read More

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http://link.springer.com/10.1007/s13311-018-0661-0
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http://dx.doi.org/10.1007/s13311-018-0661-0DOI Listing
January 2019
4 Reads

Inhibition of miR-155 Limits Neuroinflammation and Improves Functional Recovery After Experimental Traumatic Brain Injury in Mice.

Neurotherapeutics 2019 Jan;16(1):216-230

Department of Anesthesiology and Shock, Trauma and Anesthesiology Research (STAR) Center, University of Maryland School of Medicine, Baltimore, MD, USA.

Micro-RNAs (miRs) are short, noncoding RNAs that negatively regulate gene expression at the post-transcriptional level and have been implicated in the pathophysiology of secondary damage after traumatic brain injury (TBI). Among miRs linked to inflammation, miR-155 has been implicated as a pro-inflammatory factor in a variety of organ systems. We examined the expression profile of miR-155, following experimental TBI (controlled cortical impact) in adult male C57Bl/6 mice, as well as the effects of acute or delayed administration of a miR-155 antagomir on post-traumatic neuroinflammatory responses and neurological recovery. Read More

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http://dx.doi.org/10.1007/s13311-018-0665-9DOI Listing
January 2019
3 Reads