NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics最新文献_第7页

Therapeutics Development for Spinal Muscular Atrophy 脊髓性肌萎缩症的治疗进展

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-04-01 DOI: 10.1016/j.nurx.2006.01.010

Charlotte J. Sumner

{"title":"Therapeutics Development for Spinal Muscular Atrophy","authors":"Charlotte J. Sumner","doi":"10.1016/j.nurx.2006.01.010","DOIUrl":"10.1016/j.nurx.2006.01.010","url":null,"abstract":"<div><h3>Summary</h3><p>Spinal muscular atrophy is an autosomal recessive motor neuron disease that is the leading inherited cause of infant and early childhood mortality. Spinal muscular atrophy is caused by mutation of the telomeric copy of the survival motor neuron gene (<em>SMN1</em>), but all patients retain a centromeric copy of the gene, <em>SMN2</em>. <em>SMN2</em> produces reduced amounts of full-length SMN mRNA, and spinal muscular atrophy likely results from insufficient levels of SMN protein in motor neurons. The SMN protein plays a well-established role in assembly of the spliceosome and may also mediate mRNA trafficking in the axon and nerve terminus of neurons. In patients, spinal muscular atrophy disease severity correlates inversely with increased <em>SMN2</em> gene copy number and, in transgenic mice lacking endogenous SMN, increasing <em>SMN2</em> gene copy number from two to eight prevents the SMA disease phenotype. These observations suggest that increasing SMN expression levels may be beneficial to SMA patients. Currently pursued therapeutic strategies for SMA include induction of <em>SMN2</em> gene expression, modulation of splicing of <em>SMN2-</em>derived transcripts, stabilization of SMN protein, neuroprotection of SMN deficit neurons, and <em>SMN1</em> gene replacement. Early clinical trials of candidate therapeutics are now ongoing in SMA patients. Clinical trials in this disease present a unique set of challenges, including the development of meaningful outcome measures and disease biomarkers.</p></div>","PeriodicalId":87195,"journal":{"name":"NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics","volume":"3 2","pages":"Pages 235-245"},"PeriodicalIF":0.0,"publicationDate":"2006-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.nurx.2006.01.010","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25919538","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 74

Contemporary Assessment and Pharmacotherapy of Tourette Syndrome 抽动秽语综合征的当代评价与药物治疗

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-04-01 DOI: 10.1016/j.nurx.2006.01.009

Lawrence Scahill, Gerald Erenberg, Cheston M. Berlin Jr, Cathy Budman, Barbara J. Coffey, Joseph Jankovic, Louise Kiessling, Robert A. King, Roger Kurlan, Anthony Lang, Jonathan Mink, Tanya Murphy, Samual Zinner, John Walkup

{"title":"Contemporary Assessment and Pharmacotherapy of Tourette Syndrome","authors":"Lawrence Scahill, Gerald Erenberg, Cheston M. Berlin Jr, Cathy Budman, Barbara J. Coffey, Joseph Jankovic, Louise Kiessling, Robert A. King, Roger Kurlan, Anthony Lang, Jonathan Mink, Tanya Murphy, Samual Zinner, John Walkup","doi":"10.1016/j.nurx.2006.01.009","DOIUrl":"10.1016/j.nurx.2006.01.009","url":null,"abstract":"<div><h3>Summary</h3><p>To develop a guide to clinical assessment and pharmacotherapy for children and adults with Tourette syndrome (TS), we reviewed published literature over the past 25 years to identify original articles and reviews on the assessment and pharmacological treatment of Tourette syndrome, attention–deficit/hyperactivity disorder (ADHD) and obsessive–compulsive disorder (OCD). The literature search also included a survey of reviews published in book chapters. The assessment section was compiled from several reviews. Pharmacological treatments were classified into those with strong empirical support (as evidenced by two positive placebo-controlled studies for tics, OCD, or ADHD in TS samples); modest empirical support (one positive placebo-controlled study), or minimal support (open-label data only). We conclude that accurate diagnosis, including identification of comorbid conditions, is an essential step toward appropriate treatment for patients with TS. In many patients with TS, symptom management requires pharmacotherapy for tics or coexisting conditions. The evidence supporting efficacy and safety for medications used in patients with TS varies. But this evidence offers the best guide to clinical practice.</p></div>","PeriodicalId":87195,"journal":{"name":"NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics","volume":"3 2","pages":"Pages 192-206"},"PeriodicalIF":0.0,"publicationDate":"2006-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.nurx.2006.01.009","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25920169","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 252

Therapeutic Interventions for Tone Abnormalities in Cerebral Palsy 脑瘫患者音调异常的治疗干预

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-04-01 DOI: 10.1016/j.nurx.2006.01.008

Ann H. Tilton

引用次数: 39

Pediatric Neurotherapy 儿科神经病治疗

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-04-01 DOI: 10.1016/j.nurx.2006.02.001

Leon S. Dure IV M.D. (Guest Editor), Faye Silverstein M.D. (Guest Editor)

引用次数: 0

New Generation Anticonvulsants for the Treatment of Epilepsy in Children 用于治疗儿童癫痫的新一代抗惊厥药物。

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-04-01 DOI: 10.1016/j.nurx.2006.01.013

Elizabeth J. Donner, O. Carter Snead III

引用次数: 32

The Congenital Disorders of Glycosylation: A Multifaceted Group of Syndromes 先天性糖基化疾病:一组多方面的综合征

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-04-01 DOI: 10.1016/j.nurx.2006.01.012

Erik A. Eklund , Hudson H. Freeze

引用次数: 65

Therapeutic Approaches and Advances in Pediatric Stroke 儿童中风的治疗方法和进展

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-04-01 DOI: 10.1016/j.nurx.2006.01.003

Adam Kirton MD, MSc (FRCPC), Gabrielle deVeber MD, MHSc (FRCPC)

引用次数: 15

Advances in the Treatment of Anxiety: Targeting Glutamate 以谷氨酸为靶点的焦虑治疗进展

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-01-01 DOI: 10.1016/j.nurx.2005.12.005

Asher B. Simon, Jack M. Gorman

引用次数: 44

Neural Circuitry and Neuroplasticity in Mood Disorders: Insights for Novel Therapeutic Targets 情绪障碍中的神经回路和神经可塑性:对新的治疗靶点的见解

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-01-01 DOI: 10.1016/j.nurx.2005.12.009

Paul J. Carlson , Jaskaran B. Singh , Carlos A. Zarate Jr , Wayne C. Drevets , Husseini K. Manji

{"title":"Neural Circuitry and Neuroplasticity in Mood Disorders: Insights for Novel Therapeutic Targets","authors":"Paul J. Carlson , Jaskaran B. Singh , Carlos A. Zarate Jr , Wayne C. Drevets , Husseini K. Manji","doi":"10.1016/j.nurx.2005.12.009","DOIUrl":"10.1016/j.nurx.2005.12.009","url":null,"abstract":"<div><h3>Summary</h3><p>Major depressive disorder and bipolar disorder are severe mood disorders that affect the lives and functioning of millions each year. The majority of previous neurobiological research and standard pharmacotherapy regimens have approached these illnesses as purely neurochemical disorders, with particular focus on the monoaminergic neurotransmitter systems. Not altogether surprisingly, these treatments are inadequate for many individuals afflicted with these devastating illnesses. Recent advances in functional brain imaging have identified critical neural circuits involving the amygdala and other limbic structures, prefrontal cortical regions, thalamus, and basal ganglia that modulate emotional behavior and are disturbed in primary and secondary mood disorders. Growing evidence suggests that mechanisms of neural plasticity and cellular resilience, including impairments of neurotrophic signaling cascades as well as altered glutamatergic and glucocorticoid signaling, underlie the dysregulation in these circuits. The increasing ability to monitor and modulate activity in these circuits is beginning to yield greater insight into the neurobiological basis of mood disorders. Modulation of dysregulated activity in these affective circuits via pharmacological agents that enhance neuronal resilience and plasticity, and possibly via emerging nonpharmacologic, circuitry-based modalities (for example, deep brain stimulation, magnetic stimulation, or vagus nerve stimulation) offers promising targets for novel experimental therapeutics in the treatment of mood disorders.</p></div>","PeriodicalId":87195,"journal":{"name":"NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics","volume":"3 1","pages":"Pages 22-41"},"PeriodicalIF":0.0,"publicationDate":"2006-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.nurx.2005.12.009","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25866380","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 103

Advances in the Treatment of Depression 抑郁症治疗的进展

NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics Pub Date : 2006-01-01 DOI: 10.1016/j.nurx.2005.12.007

Paul E. Holtzheimer III, Charles B. Nemeroff

{"title":"Advances in the Treatment of Depression","authors":"Paul E. Holtzheimer III, Charles B. Nemeroff","doi":"10.1016/j.nurx.2005.12.007","DOIUrl":"10.1016/j.nurx.2005.12.007","url":null,"abstract":"<div><h3>Summary</h3><p>Depression is a highly prevalent and disabling condition associated with significant morbidity and mortality. Currently available treatments for depression include tricyclic antidepressants, monoamine oxidase inhibitors, selective serotonin reuptake inhibitors, serotonin norepinephrine reuptake inhibitors, various atypical antidepressants, and electroconvulsive therapy. Although these treatments are effective, a significant number of patients do not respond or achieve sustained remission despite aggressive management. Advances in the neurobiology of depression have suggested a number of novel targets for antidepressant treatment. Based on an improved understanding of the neurobiology of depression, several novel pharmacologic and nonpharmacologic interventions are being developed. Pharmacologic developments include CRF antagonists, glucocorticoid receptor antagonists, substance P receptor antagonists, NMDA glutamate receptor antagonists, transdermal selegiline, so-called “triple” reuptake inhibitors, and augmentation of typical antidepressant medications with atypical antipsychotics. Nonpharmacologic advances have largely involved focal brain stimulation techniques including vagus nerve stimulation, transcranial magnetic stimulation, magnetic seizure therapy, and deep brain stimulation. For the most part, the data on these treatments are preliminary, and more study is needed to clarify their potential clinical benefit. However, it is clear that further study of the neurobiology of depression will continue to provide a rationale for developing innovative targets for antidepressant therapies.</p></div>","PeriodicalId":87195,"journal":{"name":"NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics","volume":"3 1","pages":"Pages 42-56"},"PeriodicalIF":0.0,"publicationDate":"2006-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.nurx.2005.12.007","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25866381","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 54