Pharmacological therapeutics in Friedreich ataxia: the present state.

Abstract:

INTRODUCTION:Friedreich ataxia (FRDA) is a progressive, inherited, neurodegenerative disease for which there is currently no cure or approved treatment. FRDA is caused by deficits in the production and expression of frataxin, a protein found in the mitochondria that is most likely responsible for regulating iron-sulfur cluster enzymes within the cell. A decrease in frataxin causes dysfunction of adenosine triphosphate synthesis, accumulation of mitochondrial iron, and other events leading to downstream cellular dysfunction. Areas covered: Therapeutic development for FRDA currently focuses on improving mitochondrial function and finding ways to increase frataxin expression. Additionally, the authors will review potential approaches aimed at iron modulation and genetic modulation. Finally, gene therapy is progressing rapidly and is being explored as a treatment for FRDA. Expert commentary: The collection of multiple therapeutic approaches provides many possible ways to treat FRDA. Although the mitochondrial approaches are not thought to be curative, as the primary frataxin deficit will remain, they may still produce improvements in quality of life and slowing of progression. Therapies aimed at frataxin restoration are more likely to truly modify the disease, with gene therapy as the best possibility to alter the course of the disease from both a cardiac and neurological perspective.

journal_name

Expert Rev Neurother

authors

Strawser C,Schadt K,Hauser L,McCormick A,Wells M,Larkindale J,Lin H,Lynch DR

doi

10.1080/14737175.2017.1356721

subject

Has Abstract

pub_date

2017-09-01 00:00:00

pages

895-907

issue

9

eissn

1473-7175

issn

1744-8360

journal_volume

17

pub_type

杂志文章,评审
  • Dual tasking in Parkinson's disease: should we train hazardous behavior?

    abstract::Dual-task (DT) circumstances aggravate gait disorders in Parkinson's disease (PD) and are associated with an increased risk of falling and reduced functional mobility. Clinical rehabilitation guidelines for PD consider DT interventions as potentially hazardous and recommend avoiding them in daily life. The current art...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.2015.1077116

    authors: Strouwen C,Molenaar EA,Münks L,Keus SH,Bloem BR,Rochester L,Nieuwboer A

    更新日期:2015-01-01 00:00:00

  • Comparing treatment options for chronic inflammatory neuropathies and choosing the right treatment plan.

    abstract:INTRODUCTION:Chronic inflammatory neuropathies are disorders caused by an immune response to peripheral nerve. They include chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), multifocal motor neuropathy (MMN) and neuropathy associated with anti-MAG IgM monoclonal gammopathy and other less frequent neurop...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1080/14737175.2017.1340832

    authors: Nobile-Orazio E,Gallia F,Terenghi F,Bianco M

    更新日期:2017-08-01 00:00:00

  • Use of neural stem cells as therapeutic vehicles for the treatment of malignant glioma.

    abstract::The prognosis for patients with malignant glioma, the most common primary intracranial neoplasm, remains dismal despite significant progress in neuro-oncological therapies and technology. This stems from the inability of current treatment strategies to address the highly invasive nature of this disease. Malignant glia...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章

    doi:10.1586/14737175.3.6.883

    authors: Ehtesham M,Kabos P,Black KL,Yu JS

    更新日期:2003-11-01 00:00:00

  • Light and chemical control of neuronal circuits: possible applications in neurotherapy.

    abstract::Millions of people worldwide suffer from diseases that result from a failure of central pathways to regulate behavioral and physiological processes. Advances in genetics and pharmacology have already allowed us to appreciate that rather than this dysregulation being systemic throughout the brain, it is usually rooted ...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.2014.948850

    authors: Whittle AJ,Walsh J,de Lecea L

    更新日期:2014-09-01 00:00:00

  • Pharmacotherapy for social anxiety disorder: a systematic review.

    abstract::Social anxiety disorder (SAD) is a prevalent, disabling disorder. We aimed to assess the effects of pharmacotherapy for SAD and to determine whether particular classes of medication are more effective and/or better tolerated than others. A systematic review and meta-analysis was conducted of all published and unpublis...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.8.2.235

    authors: Ipser JC,Kariuki CM,Stein DJ

    更新日期:2008-02-01 00:00:00

  • Recent genetic advances in ADHD and diagnostic and therapeutic prospects.

    abstract::Attention deficit hyperactivity disorder (ADHD) is a common behavioral disorder of a complex nature. Genetic and environmental factors are thought to be involved in precipitating the disorder. Pharmacological, animal model and recent molecular studies support the role of genes (of minor or medium effect) from dopamine...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章

    doi:10.1586/14737175.3.4.453

    authors: Hawi Z,Kirley A,Lowe N,Fitzgerald M,Gill M

    更新日期:2003-07-01 00:00:00

  • New advances in the rehabilitation of CNS diseases applying rTMS.

    abstract::Transcranial magnetic stimulation (TMS) can directly stimulate the CNS, modifying the brain's plasticity to enhance the behavior of the paretic extremities. Studies with low-frequency repetitive TMS (rTMS) on the intact hemisphere and those with high frequencies on the affected hemisphere could increase the speed of m...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.7.2.165

    authors: Málly J,Stone TW

    更新日期:2007-02-01 00:00:00

  • Rivastigmine for Alzheimer's disease.

    abstract::Alzheimer's disease is the most common form of neurodegenerative dementia and poses considerable health challenges to both patients and their families. Rivastigmine is a powerful slow-reversible, noncompetitive carbamate cholinesterase inhibitor that is approved for the treatment of mild-to-moderate Alzheimer's diseas...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.5.5.563

    authors: Desai AK,Grossberg GT

    更新日期:2005-09-01 00:00:00

  • Cardiovascular effects of antipsychotics.

    abstract::There is great concern over cardiovascular disease in the schizophrenic population owing to the high incidence of cardiovascular mortality. Increased cardiovascular mortality is related to lifestyle choices (e.g., smoking and sedentary lifestyle) and a high prevalence of comorbid medical conditions, including dyslipid...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.7.7.829

    authors: Michelsen JW,Meyer JM

    更新日期:2007-07-01 00:00:00

  • Pharmacological approaches for Alzheimer's disease: neurotransmitter as drug targets.

    abstract::Alzheimer's disease (AD) is the most common CNS disorder occurring worldwide. There is neither proven effective prevention for AD nor a cure for patients with this disorder. Hence, there is an urgent need to develop safer and more efficacious drugs to help combat the tremendous increase in disease progression. The pre...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.2015.988709

    authors: Prakash A,Kalra J,Mani V,Ramasamy K,Majeed AB

    更新日期:2015-01-01 00:00:00

  • CSD, BBB and MMP-9 elevations: animal experiments versus clinical phenomena in migraine.

    abstract::Cortical spreading depression (CSD) has been at the center stage of migraine pathophysiology for approximately six decades. Reanalysis of CSD reveals several major unbridgeable gaps in this experimental neurophysiologic concept for migraine. Key phenotypic and pharmacological features of migraine challenge the assumed...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/ern.09.103

    authors: Gupta VK

    更新日期:2009-11-01 00:00:00

  • Interventions to promote the evidence-based care of children with ADHD in primary-care settings.

    abstract::Attention-deficit/hyperactivity disorder (ADHD) is a commonly occurring behavioral disorder among children. Community-based physicians are often the primary providers of services for children with ADHD. A set of consensus guidelines has been published by the American Academy of Pediatrics that provides best-practice d...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/ern.09.5

    authors: Langberg JM,Brinkman WB,Lichtenstein PK,Epstein JN

    更新日期:2009-04-01 00:00:00

  • Neurostimulation in primary headache syndromes.

    abstract::Neurostimulation therapy involves the use of peripheral or central nerve electrical stimulation approaches for the treatment of medically intractable headache. Currently, for peripheral stimulation the main approach is that of occipital nerve stimulation, while for central stimulation deep-brain approaches with the ta...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.7.12.1785

    authors: Goadsby PJ

    更新日期:2007-12-01 00:00:00

  • Genetics of schizophrenia: implications for treatment.

    abstract::Schizophrenia is a common, debilitating illness for which treatment is empirical and unsatisfactory. Intense efforts to identify etiological factors have been launched in order to facilitate rational therapy. Such efforts have included gene-mapping studies since a significant heritability has been proved. In common wi...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.4.4.725

    authors: Northup A,Nimgaonkar VL

    更新日期:2004-07-01 00:00:00

  • Cognitive-behavioral therapy for bipolar disorder.

    abstract::Bipolar disorder is one of the most serious and prevalent psychiatric disorders. The aim of the present article is to review the efficiency of cognitive-behavioral therapy (CBT) for bipolar patients. Some studies show consistent evidence that cognitive therapy, concomitant to psycho-education and pharmacological treat...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/ern.10.75

    authors: da Costa RT,Rangé BP,Malagris LE,Sardinha A,de Carvalho MR,Nardi AE

    更新日期:2010-07-01 00:00:00

  • Donepezil (Aricept) for treatment of Alzheimer's disease and other dementing conditions.

    abstract::Alzheimer's disease is common, incurable and disabling. It is expected to grow dramatically in prevalence over the next 50 years. At current, the standard of care for patients with mild and moderately severe Alzheimer's disease includes the use of acetylcholinesterase inhibitors. Donepezil (Aricept) is a highly select...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.4.1.5

    authors: Geldmacher DS

    更新日期:2004-01-01 00:00:00

  • Systemic thrombolytic therapy of acute ischemic stroke with rtPA.

    abstract::Systemic thrombolysis with recombinant tissue plasminogen activator is the first therapy proven to be effective in acute ischemic stroke. Nevertheless, its efficacy and feasibility are still a matter of debate. Much of this debate derives from uncertainties regarding patient eligibility and problems implementing this ...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章

    doi:10.1586/14737175.2.2.187

    authors: Koennecke HC

    更新日期:2002-03-01 00:00:00

  • Treatment of neoplastic meningitis: what is the standard of care?

    abstract::This discussion will focus on defining the standard of care for neoplastic meningitis, including: What is the role of radiation therapy? What are first-line pharmacotherapeutic agents? Is combination therapy useful? What is the role of corticosteroids? ...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.4.4.S11

    authors: Recht L,Phuphanich S

    更新日期:2004-07-01 00:00:00

  • Molecularly targeted therapies for malignant glioma: rationale for combinatorial strategies.

    abstract::Median survival of patients with malignant glioma (MG) from time of diagnosis is approximately 1 year, despite surgery, irradiation and conventional chemotherapy. Improving patient outcome relies on our ability to develop more effective therapies that are directed against the unique molecular aberrations within a pati...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/ern.09.116

    authors: Thaker NG,Pollack IF

    更新日期:2009-12-01 00:00:00

  • Retinoic acid as a therapeutic option in Alzheimer's disease: a focus on cholinergic restoration.

    abstract::Retinoic acid is a potent cell differentiating factor, which through its nuclear receptors affects a vast range of promoter sites in brain neuronal and glial cells in every step of embryonic and postnatal life. Its capacities, facilitating maturation of neurotransmitter phenotype in different groups of neurons, pave t...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.2015.1008456

    authors: Szutowicz A,Bielarczyk H,Jankowska-Kulawy A,Ronowska A,Pawełczyk T

    更新日期:2015-03-01 00:00:00

  • Voxel-based morphometry in Alzheimer's disease.

    abstract::Recent morphometric MRI studies have investigated brain volume abnormalities associated with the diagnosis of Alzheimer's disease (AD) using voxel-based morphometry (VBM). This technique allows the assessment of gray matter volumes in subjects with AD or related conditions compared with healthy controls in an automate...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.8.11.1691

    authors: Busatto GF,Diniz BS,Zanetti MV

    更新日期:2008-11-01 00:00:00

  • Menstrual migraine: clinical and therapeutical aspects.

    abstract::Estrogens fluctuations, particularly their premenstrual fall, are currently regarded as the main triggers of menstrual migraine (MM). MM presents in two clinical forms: pure MM, where attacks are confined to the perimenstrual period (PMP), and menstrually related migraine, where attacks always occur during, but are no...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.7.9.1105

    authors: Allais G,Castagnoli Gabellari I,De Lorenzo C,Mana O,Benedetto C

    更新日期:2007-09-01 00:00:00

  • Role of homocysteine in the treatment of Parkinson's disease.

    abstract::The saga of harmful administration of levodopa (LD) in the treatment of Parkinson's disease (PD) resulted from outcomes of animal trials and cell culture studies. They were initiated after the clinical observation of onset of motor complications related to the short plasma half-life of the drug in PD patients. This di...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.8.6.957

    authors: Müller T

    更新日期:2008-06-01 00:00:00

  • Rufinamide: a new antiepileptic drug treatment for Lennox-Gastaut syndrome.

    abstract::Lennox-Gastaut syndrome is a relatively rare epilepsy syndrome that usually begins in early-mid childhood and is characterized by multiple seizure types, particularly generalized seizures, which are often resistant to antiepileptic drug medication. Rufinamide is a new antiepileptic drug approved as adjunctive therapy ...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/ern.10.51

    authors: Ferrie CD

    更新日期:2010-06-01 00:00:00

  • Depression and randomized, controlled trials: problems and solutions.

    abstract::Research into what works in depression is in trouble. All treatments appear equally effective and what happens in research trials does not seem to translate into clinical practice or, to put it another way, clinical practice is not reflected in randomized controlled trials. We run the risk of designing cosmetic trials...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.6.1.41

    authors: Hatcher S

    更新日期:2006-01-01 00:00:00

  • Treatment of motor fluctuations in Parkinson's disease: recent developments and future directions.

    abstract::Parkinson's disease (PD) is characterized clinically by rest tremor, rigidity, bradykinesia and pathologically by degeneration of nigrostriatal dopamine neurons. Motor fluctuations (wearing off) and motor complications (dyskinesia) are common features of the long-term treatment of PD. Ongoing clinical and preclinical ...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.2014.868306

    authors: Ramirez-Zamora A,Molho E

    更新日期:2014-01-01 00:00:00

  • The need for emergency treatment of transient ischemic attack and minor stroke.

    abstract::The risk of recurrent stroke following transient ischemic attack or minor stroke has recently been shown to be 5-10% at 1 week and 10-20% at 3 months, depending on study population and methods. This is considerably higher than previously estimated and current clinical guidelines reflect the need for rapid assessment a...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.5.2.203

    authors: Giles MF,Rothwell PM

    更新日期:2005-03-01 00:00:00

  • Supportive management of patients with brain tumors.

    abstract::Management issues for patients with brain tumors include peritumoral edema, symptomatic seizures, venous thromboembolism, headache, pain, fatigue and neuropsychological complaints. Vasogenic edema is typically ameliorated with the lowest dose possible of corticosteroid. Seizures are managed with attention to additiona...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.7.10.1327

    authors: Daly FN,Schiff D

    更新日期:2007-10-01 00:00:00

  • Association of chronic divalproex sodium use and brain atrophy in Alzheimer's disease.

    abstract::Divalproex sodium has been widely use for the treatment of bipolar disorder, behavioral control in schizophrenia, seizure and agitation in Alzheimer's disease. With the advent of other mood stabilizers and anticonvulsants, the use of divaplroex sodium has been slightly decreased; however, it has been a major mediation...

    journal_title:Expert review of neurotherapeutics

    pub_type: 评论,杂志文章

    doi:10.1586/ern.11.194

    authors: Han C,Shim DS,Lee SJ,Patkar AA,Masand PS,Pae CU

    更新日期:2012-02-01 00:00:00

  • Effectiveness of paroxetine in the treatment of obsessive-compulsive disorders.

    abstract::Clomipramine ushered in a new age of pharmacotherapy for obsessive-compulsive disorders, and it also facilitated our understanding of the biological aspects of obsessive-compulsive disorder, focusing on the serotonergic systems. The introduction of selective serotonin reuptake inhibitors has led to great progress in t...

    journal_title:Expert review of neurotherapeutics

    pub_type: 杂志文章,评审

    doi:10.1586/14737175.6.7.945

    authors: Kamijima K,Aoki M

    更新日期:2006-07-01 00:00:00