Abstract:
:Spinal muscular atrophy (SMA) is an autosomal recessive disease characterized by degeneration of spinal cord motor neurons and muscular atrophy. Advances in recent research have led to understanding of the molecular genetics of SMA. Therapeutic strategies have been developed according to the unique genomic structure of the SMN genes. Three groups of compounds have been identified as therapeutic candidates. One group was identified before the molecular genetics of SMA was understood, chosen on the basis of their effectiveness in similar neurologic disorders. The second group was identified based on their ability to modify SMN2 gene expression. Several of these agents are currently in clinical trials. A third group, identified by large-scale drug screening, is still under preclinical investigation. In addition, other advances in medical technology have led to the publication of a consensus statement regarding the care of SMA patients.
journal_name
Curr Treat Options Neuroljournal_title
Current treatment options in neurologyauthors
Wang CH,Lunn MRdoi
10.1007/s11940-008-0044-7subject
Has Abstractpub_date
2008-11-01 00:00:00pages
420-8issue
6eissn
1092-8480issn
1534-3138journal_volume
10pub_type
杂志文章abstract:PURPOSE OF REVIEW:The purpose of this review is to summarize the current evidence regarding the impact of the exposure to anesthetic and sedative agents on neurodevelopment during the period of rapid brain growth in the first 3 years of life. Though much of the definitive data demonstrating anesthesia-induced neurotoxi...
journal_title:Current treatment options in neurology
pub_type: 杂志文章,评审
doi:10.1007/s11940-018-0536-z
更新日期:2018-10-13 00:00:00
abstract::Medical management, the mainstay of treatment for partial epilepsy, involves choosing from an array of antiseizure drugs, most of which have comparable efficacy against partial-onset seizures. Effective treatment strategies involve matching the characteristics of drug and patient to control seizures without adverse ef...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-009-0028-2
更新日期:2009-07-01 00:00:00
abstract:OPINION STATEMENT:Paraneoplastic brain stem encephalitis can occur as an isolated clinical syndrome or, more often, may be part of a more widespread encephalitis. Different antineuronal autoantibodies, such as anti-Hu, anti-Ri, and anti-Ma2 can be associated with the syndrome, and the most frequent tumors are lung and ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-013-0221-1
更新日期:2013-04-01 00:00:00
abstract::Lysosomal storage disorders (LSDs), over 40 different diseases, are now considered treatable disorders. Only a few short years ago, Lysosomal storage disorders were seen as interesting neurodegenerative disorders without any potential for treatment. Effective treatment strategies such as bone marrow transplantation (B...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-001-0006-9
更新日期:2001-05-01 00:00:00
abstract::Amyotrophic lateral sclerosis (ALS) is a progressive motor neuron disease that usually results in death secondary to respiratory failure. The psychological reaction to the diagnosis of ALS in patient and caregiver has received increasing attention. Reports are highly variable as to the severity of depressive symptoms ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-006-0030-x
更新日期:2006-09-01 00:00:00
abstract::Sleep disorders can have a significant impact on the growth and development of children. Behavioral intervention is the first step in the management of many sleep disorders. Limit-setting is particularly challenging in younger children but must be consistent. Good sleep hygiene practices must be emphasized to adolesce...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-007-0042-1
更新日期:2007-11-01 00:00:00
abstract::Metabolic myopathies are disorders affecting utilization of carbohydrates or fat in the skeletal muscle. Adult patients with metabolic myopathies typically present with exercise-induced pain, contractures or stiffness, fatigue, and myoglobinuria. Symptoms are related to energy failure. Purpose of review In this review...
journal_title:Current treatment options in neurology
pub_type: 杂志文章,评审
doi:10.1007/s11940-017-0473-2
更新日期:2017-09-21 00:00:00
abstract::The epidemiologic link between increased low-density-lipoprotein (LDL) cholesterol and ischemic stroke is controversial. However, recent trials make it clear that LDL reduction by statin agents in high-risk patients reduces the risk of ischemic stroke. The risk of first stroke is reduced within 1 to 2 years of statin ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-006-0039-1
更新日期:2006-11-01 00:00:00
abstract::OPINION STATEMENT: Anemia develops in about 50% of patients hospitalized with traumatic brain injury (TBI) and is recognized as a cause of secondary brain injury. This review examines the effects of anemia and transfusion on TBI patients through a literature search to identify original research on anemia and transfusi...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-012-0167-8
更新日期:2012-04-01 00:00:00
abstract::Migraine is a chronic paroxysmal neurological disorder characterized by multiphase attacks of head pain and a myriad of neurological symptoms. Chronic migraine causes a great personal and societal burden. Many patients are poorly responsive to, or non-compliant with, conventional migraine preventive therapies. For thi...
journal_title:Current treatment options in neurology
pub_type: 杂志文章,评审
doi:10.1007/s11940-019-0555-4
更新日期:2019-03-14 00:00:00
abstract:OPINION STATEMENT:Narcolepsy is a severe, incurable, neurological disorder that is treated by pharmacological management of its symptoms. The main symptoms are excessive daytime sleepiness (EDS) and cataplexy, although addition symptoms that may require treatment include sleep paralysis, hypnagogic hallucinations, and ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-015-0347-4
更新日期:2015-05-01 00:00:00
abstract::The neurorehabilitation program for cerebral palsy changes over time. During the first 2 years of life, an infant stimulation program with an emphasis on more than just improving motor deficits is emphasized. The importance of involvement of a knowledgeable therapist cannot be overemphasized. Realistic expectations mu...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-000-0011-4
更新日期:2000-03-01 00:00:00
abstract:OPINION STATEMENT:Atrial fibrillation (AF) is the most common sustained cardiac rhythm disorder, which can potentially increases the risk of stroke by five-fold, thus, resulting in high public healthcare burden. Stroke prevention is vital in the management of AF patients. Vitamin K antagonists (VKA, eg, warfarin) have ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-014-0331-4
更新日期:2015-02-01 00:00:00
abstract:OPINION STATEMENT:Autoimmune neuromuscular disorders in childhood include Guillain-Barré syndrome and its variants, chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), juvenile myasthenia gravis (JMG), and juvenile dermatomyositis (JDM), along with other disorders rarely seen in childhood. In general, the...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-011-0146-5
更新日期:2011-12-01 00:00:00
abstract:OPINION STATEMENT:Magnetic resonance imaging (MRI) is the gold standard guiding diagnostic and therapeutic management in glioma with its high resolution and possibility to depict blood-brain-barrier disruption when contrast medium is applied. In light of the shifting paradigms revealing distinct tumor subtypes based on...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-015-0384-z
更新日期:2016-01-01 00:00:00
abstract::Apathy is a common problem observed in numerous neurologic, psychiatric, and other medical conditions. It is independent from other symptoms and has been associated with a variety of negative outcomes. However, little empirical research has been conducted on potential psychologic or pharmacologic treatments for apathy...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:
更新日期:2007-09-01 00:00:00
abstract:OPINION STATEMENT:Hyponatremia is the most common electrolyte abnormality in both inpatient and outpatient settings. The condition primarily results from the combination of impaired free water excretion due to elevated vasopressin levels in conjunction with a source of free water intake. Recent studies have revealed th...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-014-0310-9
更新日期:2014-09-01 00:00:00
abstract:OPINION STATEMENT:A patient presenting with suspected primary central nervous system (CNS) vasculitis is often difficult to manage. Once the diagnosis is suspected, the clinical course may already be severe and warrant timely aggressive treatment with potentially toxic drugs. Definitive diagnosis is often delayed, requ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-013-0227-8
更新日期:2013-06-01 00:00:00
abstract::The challenge for the neurologist in the treatment of Lyme neuroborreliosis is not in the treatment per se, but in the diagnosis. Neurological manifestations of Lyme disease can present in many forms, and diagnostic techniques which detect the spirochete directly; the culture or polymerase chain reaction of the spiroc...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-005-0026-y
更新日期:2005-03-01 00:00:00
abstract:OPINION STATEMENT:The timely implementation of immunotherapy is key to successful treatment of autoimmune encephalopathies or dementias (from here on will be referred to as autoimmune encephalopathies). There are different levels of diagnostic certainty which should guide the immunological treatment of autoimmune encep...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-013-0251-8
更新日期:2013-12-01 00:00:00
abstract::Hemifacial spasm (HFS) is a peripheral movement disorder caused by direct or indirect compression or distortion of the root exit zone of the seventh cranial nerve, which is most commonly compressed by an arterial loop, but also may be compressed by a tumor, cyst, or aneurysm. All patients with HFS should undergo magne...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-004-0009-4
更新日期:2004-05-01 00:00:00
abstract::Multiple pharmacologic treatments have been studied, but propranolol and primidone have proven to be the most effective medications. It is unlikely that a patient will respond miraculously to another medication if his or her response to propranolol and primidone is minimal. Some subsets of essential tremor, however, s...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-999-0027-3
更新日期:1999-03-01 00:00:00
abstract:OPINION STATEMENT:Triptans, selective serotonin receptor agonists, were developed almost 20 years ago and represented a major breakthrough in the treatment of acute migraine attacks. Since then, the triptans have become first-line agents for most patients with migraine, both with and without aura, unless contraindicate...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-010-0082-9
更新日期:2010-09-01 00:00:00
abstract:OPINION STATEMENT:Melanoma-associated retinopathy is a rare paraneoplastic disorder that is challenging to diagnose and even more difficult to treat. Because of the rarity of the disease, therapy is based on analysis of case series and case reports. Based on evidence from these reports, first-line therapy is cytoreduct...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-009-0057-x
更新日期:2010-01-01 00:00:00
abstract:OPINION STATEMENT:Restless Legs Syndrome/Willis-Ekbom Disease (RLS/WED) is a common condition characterized by an irresistible urge to move the legs, concomitant with an unpleasant sensation in the lower limbs, which is typically relieved by movement. Symptoms occur predominantly at rest and prevail in the afternoon or...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-015-0390-1
更新日期:2016-02-01 00:00:00
abstract:OPINION STATEMENT:Paroxysmal dyskinesia represents a group of uncommon movement disorders that are characterized by episodes of abnormal movements arising from a baseline of normal or nearly normal movement. Recent advances in the genetics of these disorders have helped provide some unification of classification scheme...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-015-0350-9
更新日期:2015-06-01 00:00:00
abstract::Respiratory complications account for the majority of deaths occurring in patients suffering from amyotrophic lateral sclerosis (ALS). Patients normally succumb to their illness within an average of 3 to 5 years from the time of diagnosis from complications such as hypoventilation, hypoxemia, hypercarbia, aspiration, ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-001-0048-z
更新日期:2001-03-01 00:00:00
abstract:OPINION STATEMENT:Parkinson's disease (PD) has been increasingly recognized as having a multitude of nonmotor symptoms including psychosis, cognitive impairment and dementia, mood disturbances, fatigue, apathy, and sleep disorders. Psychosis and dementia, in particular, greatly affect quality of life for both patients ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-013-0281-2
更新日期:2014-03-01 00:00:00
abstract::Antiepileptic drug (AED) treatment is associated with multiple short- and long-term side effects. Effects on endocrine function, including weight change, reproductive function, thyroid function, and bone health are examples of these side effects. Some AEDs affect weight, resulting in weight gain or loss. Levetiracetam...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-005-0037-8
更新日期:2005-07-01 00:00:00
abstract:OPINION STATEMENT:Pervasive developmental disorders (PDDs), including autistic disorder (autism), Asperger's disorder, and pervasive developmental disorder not otherwise specified (PDD NOS), are neurodevelopmental disorders of childhood onset. These disorders persist throughout the lifespan of affected individuals and ...
journal_title:Current treatment options in neurology
pub_type: 杂志文章
doi:10.1007/s11940-010-0091-8
更新日期:2010-11-01 00:00:00