☆ 4.6 Review

Pharmacotherapy for Amyotrophic Lateral Sclerosis: A Review of Approved and Upcoming Agents

DRUGS (2022)

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Article Clinical Neurology

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

Sabrina Paganoni et al.

Summary: The current therapeutic development in ALS faces limitations in terms of cost, time, and flexibility due to individual randomized clinical trials. Adaptive platform trials provide a novel approach to investigate multiple interventions for a single disease continuously. The Healey ALS Platform Trial is a recently launched trial that aims to identify novel treatments, biomarkers, and trial endpoints rapidly by testing multiple investigational products concurrently in ALS patients.

ANNALS OF NEUROLOGY (2022)

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Article Clinical Neurology

A randomized placebo-controlled phase 3 study of mesenchymal stem cells induced to secrete high levels of neurotrophic factors in amyotrophic lateral sclerosis

Merit E. Cudkowicz et al.

Summary: This study evaluated the efficacy of mesenchymal stem cells induced to secrete neurotrophic factors in slowing the progression of ALS. The results suggest that this therapy may have a positive effect on patients with less severe disease, but did not reach statistical significance on the primary endpoint.

MUSCLE & NERVE (2022)

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Article Biochemistry & Molecular Biology

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

Vladislav A. Korobeynikov et al.

Summary: This study demonstrates that an antisense oligonucleotide can effectively lower the levels of FUS protein in mice and in a patient with FUS-dependent ALS. The research provides evidence that the insolubility of FUS and related RNA-binding proteins contribute to neurodegeneration in ALS-FTD. Silencing FUS using a non-allele-specific antisense oligonucleotide delays motor neuron degeneration in a disease-relevant mouse model and reduces the burden of FUS aggregates in ALS patients.

NATURE MEDICINE (2022)

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Article Clinical Neurology

Safety and Effectiveness of Long-term Intravenous Administration of Edaravone for Treatment of Patients With Amyotrophic Lateral Sclerosis

Simon Witzel et al.

Summary: This study found that long-term intravenous edaravone therapy for patients with ALS was feasible and well tolerated, but did not provide any disease-modifying benefit compared to standard therapy.

JAMA NEUROLOGY (2022)

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Article Multidisciplinary Sciences

TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A

Anna-Leigh Brown et al.

Summary: Risk variants in the synaptic gene UNC13A are associated with increased risk of ALS and FTD. These variants lead to the inclusion of a cryptic exon in UNC13A when TDP-43 is depleted, resulting in the loss of UNC13A protein. Two common UNC13A polymorphisms strongly associated with ALS and FTD risk overlap with TDP-43 binding sites.

NATURE (2022)

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Article Multidisciplinary Sciences

TDP-43 represses cryptic exon inclusion in the FTD-ALS gene UNC13A

X. Rosa Ma et al.

Summary: A hallmark feature of ALS and FTD is the depletion of RNA-binding protein TDP-43 from neurons, and a major function of TDP-43 is to repress cryptic exon inclusion. UNC13A gene polymorphisms are strongly associated with FTD and ALS, and this study shows that loss of TDP-43 leads to cryptic exon splicing in UNC13A. The top genetic risk factors for FTD and ALS (UNC13A genetic variants) are shown to be functionally linked to TDP-43 dysfunction.

NATURE (2022)

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Article Clinical Neurology

Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage Amyotrophic Lateral Sclerosis A Randomized Clinical Trial

Ryosuke Oki et al.

Summary: This study aimed to validate the efficacy and safety of ultrahigh-dose methylcobalamin for patients with early-stage ALS. The results showed that methylcobalamin was effective in slowing functional decline in ALS patients, and it was safe to use during the 16-week treatment period.

JAMA NEUROLOGY (2022)

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Article Medicine, General & Internal

Motor Neuron Disease Systematic Multi-Arm Adaptive Randomised Trial (MND-SMART): a multi-arm, multi-stage, adaptive, platform, phase III randomised, double-blind, placebo-controlled trial of repurposed drugs in motor neuron disease

Charis Wong et al.

Summary: MND-SMART is a multi-arm, multi-stage, adaptive trial aimed at efficiently evaluating the efficacy of drugs. The trial design reduces time, cost, and participant numbers, while minimizing the risk of participants receiving ineffective treatments.

BMJ OPEN (2022)

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Review Cell Biology

Gene Therapy in Amyotrophic Lateral Sclerosis

Ton Fang et al.

Summary: Over 50 genes have been identified as either cause or modifier in ALS and ALS/FTD spectrum disease, with significant global efforts made to reveal biological pathways underlying the pathogenesis of these gene mutations. Various gene therapies targeting etiologic genes have been investigated and promising results have led to the application of some strategies into ALS clinical trials.

CELLS (2022)

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Article Clinical Neurology

Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis

Sabrina Paganoni et al.

Summary: In the CENTAUR trial for ALS, orally administered PB-TURSO significantly slowed functional decline and a long-term survival analysis showed that participants receiving PB-TURSO treatment had a median survival 6.5 months longer than those on placebo. These results suggest that PB-TURSO has both functional and survival benefits in ALS.

MUSCLE & NERVE (2021)

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Article Clinical Neurology

A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial OfReldesemtivIn Patients With ALS

Jeremy M. Shefner et al.

Summary: This study evaluated the safety, dose response, and preliminary efficacy of reldesemtiv in patients with ALS. Although the primary efficacy analysis did not demonstrate statistical significance, post hoc analyses showed trends favoring reldesemtiv in all endpoints, with good tolerability.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2021)

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Review Clinical Neurology

Improving clinical trial outcomes in amyotrophic lateral sclerosis

Matthew C. Kiernan et al.

Summary: Researchers emphasize that a better understanding of the biological processes in amyotrophic lateral sclerosis has led to the identification of pathogenic targets and an increasing likelihood of effective therapies. Additionally, advancements in precision medicine and improved clinical trial designs will facilitate the acceleration of therapeutic approaches.

NATURE REVIEWS NEUROLOGY (2021)

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Review Neurosciences

The genetic architecture of ALS

Aleksey Shatunov et al.

NEUROBIOLOGY OF DISEASE (2021)

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Article Medicine, General & Internal

Study protocol of RESCUE-ALS: A Phase 2, randomised, double-blind, placebo-controlled study in early symptomatic amyotrophic lateral sclerosis patients to assess bioenergetic catalysis with CNM-Au8 as a mechanism to slow disease progression

Steve Vucic et al.

Summary: Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disorder with limited treatment options. The RESCUE-ALS study investigates the potential of the novel nanocatalytic drug, CNM-Au8, to enhance metabolic and energetic capacity of motor neurones. This multicenter, randomized, double-blind study aims to evaluate the efficacy and safety of CNM-Au8 in ALS patients over a 36-week treatment period.

BMJ OPEN (2021)

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Review Neurosciences

Antibody-Based Therapeutic Interventions for Amyotrophic Lateral Sclerosis: A Systematic Literature Review

Amelie Poulin-Briere et al.

Summary: ALS is a mid-life onset neurodegenerative disease with no known cure, but antibody-based therapeutic interventions are gaining attention. Research has shown some efficacy of antibody-based treatments in ALS patients and animal models, targeting extracellular molecules and intracellular pathogenic proteins. The potentials and limitations of these therapeutic interventions have been described and discussed in the literature review.

FRONTIERS IN NEUROSCIENCE (2021)

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Review Clinical Neurology

Ataxin-2 gene: a powerful modulator of neurological disorders

Jose Miguel Laffita-Mesa et al.

Summary: Research has revealed the complex role of the Ataxin-2 gene (ATXN2) in neurological diseases, showing its involvement in various conditions such as SCA2 and peripheral nerve diseases. Understanding the mutations, genetic variants, and biological functions of ATXN2 can help in managing SCA2 and other human diseases that involve dysfunctional pathways in the brain and metabolism. Positive data from antisense oligo therapy in a murine model of SCA2 suggest potential treatment approaches for human SCA2 patients.

CURRENT OPINION IN NEUROLOGY (2021)

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Article Multidisciplinary Sciences

A RIPK1-regulated inflammatory microglial state in amyotrophic lateral sclerosis

Lauren Mifflin et al.

Summary: The study identifies a subclass of microglia, termed RIPK1-Regulated Inflammatory Microglia (RRIMs), in mouse models of ALS. RRIMs show significant up-regulation of proinflammatory pathways, are highly regulated by TNF alpha signaling, and their prevalence can be suppressed by inhibiting RIPK1 activity.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2021)

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Article Geriatrics & Gerontology

Novel STMN2 Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical Phenotype

Frances Theunissen et al.

Summary: This study identified a novel non-coding CA repeat in Stathmin-2 associated with sALS disease risk. The long/long genotype was significantly associated with disease risk, with longer CA allele length linked to earlier age-of-onset and shorter survival duration.

FRONTIERS IN AGING NEUROSCIENCE (2021)

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Article Pharmacology & Pharmacy

Bioequivalence Study of Oral Suspension and Intravenous Formulation of Edaravone in Healthy Adult Subjects

Hidetoshi Shimizu et al.

Summary: This study evaluated the bioequivalence between a 105-mg edaravone oral suspension and a 60-mg intravenous edaravone, showing similar plasma concentrations and urinary excretion of metabolites for both formulations, supporting further investigation of the oral suspension for treating amyotrophic lateral sclerosis.

CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT (2021)

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Review Neurosciences

Tweaking Progranulin Expression: Therapeutic Avenues and Opportunities

Joke Terryn et al.

Summary: Frontotemporal dementia (FTD) is a neurodegenerative disease that leads to behavioral changes and language difficulties. Mutations in progranulin (GRN) are associated with up to one-third of genetic FTD cases worldwide. Despite primarily being linked to neurodegeneration, the biological functions of GRN are diverse, ranging from wound healing to tumor cell growth.

FRONTIERS IN MOLECULAR NEUROSCIENCE (2021)

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Article Clinical Neurology

Long-term survival analysis of masitinib in amyotrophic lateral sclerosis

Jesus S. Mora et al.

Summary: The study found that oral masitinib may prolong survival in ALS patients by over 2 years compared to placebo, especially if treatment starts early and in patients with slower disease progression.

THERAPEUTIC ADVANCES IN NEUROLOGICAL DISORDERS (2021)

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Article Neuroimaging

Ibudilast (MN-166) in amyotrophic lateral sclerosis- an open label, safety and pharmacodynamic trial

Suma Babu et al.

Summary: Ibudilast is a potential neuroprotective drug that inhibits macrophage migration inhibitory factor and phosphodiesterases, but high dosage in ALS patients may lead to safety and tolerability issues.

NEUROIMAGE-CLINICAL (2021)

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Article Clinical Neurology

Masitinib as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomized clinical trial

Jesus S. Mora et al.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2020)

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Article Pharmacology & Pharmacy

A Pharmacokinetic Bioequivalence Study Comparing Sublingual Riluzole (BHV-0223) and Oral Tablet Formulation of Riluzole in Healthy Volunteers

Irfan Qureshi et al.

CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT (2020)

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Editorial Material Clinical Neurology

A proposal for new diagnostic criteria for ALSY

Jeremy M. Shefner et al.

CLINICAL NEUROPHYSIOLOGY (2020)

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Review Clinical Neurology

Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis

Jinsy A. Andrews et al.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2020)

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Article Clinical Neurology

The Italian multicenter experience with edaravone in amyotrophic lateral sclerosis

Christian Lunetta et al.

JOURNAL OF NEUROLOGY (2020)

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Article Medicine, General & Internal

SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

Christian Mueller et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

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Article Medicine, General & Internal

Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

Timothy Miller et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

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Article Medicine, General & Internal

Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis

Sabrina Paganoni et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

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Article Medicine, General & Internal

Assessment of Use and Safety of Edaravone for Amyotrophic Lateral Sclerosis in the Veterans Affairs Health Care System

Michelle Vu et al.

JAMA NETWORK OPEN (2020)

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Article Cell Biology

Targeting the Sigma-1 Receptor via Pridopidine Ameliorates Central Features of ALS Pathology in a SOD1G93A Model

Ariel Ionescu et al.

CELL DEATH & DISEASE (2019)

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Article Pharmacology & Pharmacy

Riluzole Oral Suspension: Bioavailability Following Percutaneous Gastrostomy Tube-modeled Administration Versus Direct Oral Administration

Benjamin Rix Brooks et al.

CLINICAL THERAPEUTICS (2019)

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Article Clinical Neurology

Ultra-high-dose methylcobalamin in amyotrophic lateral sclerosis: a long-term phase II/III randomised controlled study

Ryuji Kaji et al.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2019)

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Article Neurosciences

ALS-implicated protein TDP-43 sustains levels of STMN2, a mediator of motor neuron growth and repair

Joseph R. Klim et al.

NATURE NEUROSCIENCE (2019)

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Review Neurosciences

ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?

Rita Mejzini et al.

FRONTIERS IN NEUROSCIENCE (2019)

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Review Biotechnology & Applied Microbiology

AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials

Nathan Hardcastle et al.

EXPERT OPINION ON BIOLOGICAL THERAPY (2018)

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Review Clinical Neurology

Antisense oligonucleotides: the next frontier for treatment of neurological disorders

Carlo Rinaldi et al.

NATURE REVIEWS NEUROLOGY (2018)

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Article Health Care Sciences & Services

Individual treatment effect prediction for amyotrophic lateral sclerosis patients

Heidi Seibold et al.

STATISTICAL METHODS IN MEDICAL RESEARCH (2018)

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Review Biotechnology & Applied Microbiology

Advances in stem cell therapy for amyotrophic lateral sclerosis

Letizia Mazzini et al.

EXPERT OPINION ON BIOLOGICAL THERAPY (2018)

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Review Nutrition & Dietetics

How is edaravone effective against acute ischemic stroke and amyotrophic lateral sclerosis?

Kazutoshi Watanabe et al.

JOURNAL OF CLINICAL BIOCHEMISTRY AND NUTRITION (2018)

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Article Clinical Neurology

Stage at which riluzole treatment prolongs survival in patients with amyotrophic lateral sclerosis: a retrospective analysis of data from a dose-ranging study

Ton Fang et al.

LANCET NEUROLOGY (2018)

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Article Clinical Neurology

Mexiletine for muscle cramps in amyotrophic lateral sclerosis: A randomized, double-blind crossover trial

Bjorn Oskarsson et al.

MUSCLE & NERVE (2018)

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Article Medicine, General & Internal

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

M. D. Benson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

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Article Pharmacology & Pharmacy

Additional evidence for a therapeutic effect of dextromethorphan/quinidine on bulbar motor function in patients with amyotrophic lateral sclerosis: A quantitative speech analysis

Jordan R. Green et al.

BRITISH JOURNAL OF CLINICAL PHARMACOLOGY (2018)

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Article Cell & Tissue Engineering

Intraspinal Transplantation of the Adipose Tissue-Derived Regenerative Cells in Amyotrophic Lateral Sclerosis in Accordance with the Current Experts' Recommendations: Choosing Optimal Monitoring Tools

Magdalena Kuzma-Kozakiewicz et al.

STEM CELLS INTERNATIONAL (2018)

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Article Clinical Neurology

Provincial Differences in the Diagnosis and Care of Amyotrophic Lateral Sclerosis

Victoria L. Hodgkinson et al.

CANADIAN JOURNAL OF NEUROLOGICAL SCIENCES (2018)

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Article Cell Biology

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques

Florie Borel et al.

SCIENCE TRANSLATIONAL MEDICINE (2018)

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Article Chemistry, Medicinal

Riluzole 5 mg/mL oral suspension: for optimized drug delivery in amyotrophic lateral sclerosis

Ann Margaret Dyer et al.

DRUG DESIGN DEVELOPMENT AND THERAPY (2017)

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Article Clinical Neurology

Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial

Koji Abe et al.

LANCET NEUROLOGY (2017)

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Article Multidisciplinary Sciences

Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

Lindsay A. Becker et al.

NATURE (2017)

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Article Clinical Neurology

Meta-analysis of pharmacogenetic interactions in amyotrophic lateral sclerosis clinical trials

Ruben P. A. van Eijk et al.

NEUROLOGY (2017)

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Article Clinical Neurology

Enhanced Bulbar Function in Amyotrophic Lateral Sclerosis: The Nuedexta Treatment Trial

Richard Smith et al.

NEUROTHERAPEUTICS (2017)

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Article Clinical Neurology

Open-label 24-week extension study of edaravone (MCI-186) in amyotrophic lateral sclerosis

Koji Abe et al.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2017)

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Article Clinical Neurology

A post-hoc subgroup analysis of outcomes in the first phase III clinical study of edaravone (MCI-186) in amyotrophic lateral sclerosis

Koji Abe et al.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2017)

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Editorial Material Clinical Neurology

July 2017 ENCALS statement on edaravone

Ammar Al-Chalabi et al.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2017)

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Article Clinical Neurology

Tauroursodeoxycholic acid in the treatment of patients with amyotrophic lateral sclerosis

A. E. Elia et al.

EUROPEAN JOURNAL OF NEUROLOGY (2016)

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Article Clinical Neurology

Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model

Lorelei Stoica et al.

ANNALS OF NEUROLOGY (2016)

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Article Cell & Tissue Engineering

Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?

Nazem Atassi et al.

CYTOTHERAPY (2016)

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Article Medicine, General & Internal

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

Richard S. Finkel et al.

LANCET (2016)

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Review Multidisciplinary Sciences

Decoding ALS: from genes to mechanism

J. Paul Taylor et al.

NATURE (2016)

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Article Clinical Neurology

Transplantation of spinal cord-derived neural stem cells for ALS: Analysis of phase 1 and 2 trials

Jonathan D. Glass et al.

NEUROLOGY (2016)

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Article Clinical Neurology

A randomized trial of mexiletine in ALS: Safety and effects on muscle cramps and progression

Michael D. Weiss et al.

NEUROLOGY (2016)

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Article Clinical Neurology

Safety of intrathecal autologous adipose-derived mesenchymal stromal cells in patients with ALS

Nathan P. Staff et al.

NEUROLOGY (2016)

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Article Multidisciplinary Sciences

RIPK1 mediates axonal degeneration by promoting inflammation and necroptosis in ALS

Yasushi Ito et al.

SCIENCE (2016)

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Article Clinical Neurology

Riluzole exerts transient modulating effects on cortical and axonal hyperexcitability in ALS

Nimeshan Geevasinga et al.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2016)

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Review Medicine, Research & Experimental

Emerging mechanisms of molecular pathology in ALS

Owen M. Peters et al.

JOURNAL OF CLINICAL INVESTIGATION (2015)

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Article Medicine, Research & Experimental

Human neural stem cell transplantation in ALS: initial results from a phase I trial

Letizia Mazzini et al.

JOURNAL OF TRANSLATIONAL MEDICINE (2015)

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Article Clinical Neurology

Potential Environmental Factors in Amyotrophic Lateral Sclerosis

Bjoern Oskarsson et al.

NEUROLOGIC CLINICS (2015)

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Article Neurosciences

TREHALOSE DECREASES MUTANT SOD1 EXPRESSION AND ALLEVIATES MOTOR DEFICIENCY IN EARLY BUT NOT END-STAGE AMYOTROPHIC LATERAL SCLEROSIS IN A SOD1-G93A MOUSE MODEL

Y. Li et al.

NEUROSCIENCE (2015)

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Article Multidisciplinary Sciences

Edaravone, a Free Radical Scavenger, Delayed Symptomatic and Pathological Progression of Motor Neuron Disease in the Wobbler Mouse

Ken Ikeda et al.

PLOS ONE (2015)

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Review Clinical Neurology

Comprehensive rehabilitative care across the spectrum of amyotrophic lateral sclerosis

Sabrina Paganoni et al.

NEUROREHABILITATION (2015)

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Article Clinical Neurology

A single blind randomized controlled clinical trial of mexiletine in amyotrophic lateral sclerosis: Efficacy and safety of sodium channel blocker phase II trial

Kazumoto Shibuya et al.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2015)

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Article Clinical Neurology

Intraspinal neural stem cell transplantation in amyotrophic lateral sclerosis: Phase 1 trial outcomes

Eva L. Feldman et al.

ANNALS OF NEUROLOGY (2014)

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Article Biochemistry & Molecular Biology

Potential Therapeutic Drugs and Methods for the Treatment of Amyotrophic Lateral Sclerosis

G. Yacila et al.

CURRENT MEDICINAL CHEMISTRY (2014)

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Article Clinical Neurology

Analysis of amyotrophic lateral sclerosis as a multistep process: a population-based modelling study

Ammar AI-Chalabi et al.

LANCET NEUROLOGY (2014)

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Review Cell & Tissue Engineering

Concise Review: Stem Cell Therapies for Amyotrophic Lateral Sclerosis: Recent Advances and Prospects for the Future

J. Simon Lunn et al.

STEM CELLS (2014)

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Review Neurosciences

Advances in treating amyotrophic lateral sclerosis: insights from pathophysiological studies

Steve Vucic et al.

TRENDS IN NEUROSCIENCES (2014)

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Review Cell & Tissue Engineering

Therapeutic applications of mesenchymal stem cells for amyotrophic lateral sclerosis

Christina M. Lewis et al.

STEM CELL RESEARCH & THERAPY (2014)

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Article Clinical Neurology

Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients

Koji Abe et al.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2014)

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Review Clinical Neurology

Review of Dextromethorphan 20 mg/Quinidine 10mg (NUEDEXTA®) for Pseudobulbar Affect

Erik P. Pioro

NEUROLOGY AND THERAPY (2014)

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Article Biotechnology & Applied Microbiology

Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

Kevin D. Foust et al.

MOLECULAR THERAPY (2013)

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Article Clinical Neurology

Quality improvement in neurology: Amyotrophic lateral sclerosis quality measures Report of the Quality Measurement and Reporting Subcommittee of the American Academy of Neurology

Robert G. Miller et al.

NEUROLOGY (2013)

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Article Multidisciplinary Sciences

De Novo Mutations in Ataxin-2 Gene and ALS Risk

Jose Miguel Laffita-Mesa et al.

PLOS ONE (2013)

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Article Multidisciplinary Sciences

TFEB-mediated autophagy rescues midbrain dopamine neurons from α-synuclein toxicity

Mickael Decressac et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2013)

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Article Cell & Tissue Engineering

Mesenchymal stromal cell transplantation in amyotrophic lateral sclerosis: a long-term safety study

Letizia Mazzini et al.

CYTOTHERAPY (2012)

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Article Geriatrics & Gerontology

UNC13A is a modifier of survival in amyotrophic lateral sclerosis

Frank P. Diekstra et al.

NEUROBIOLOGY OF AGING (2012)

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Review Neurosciences

Viral vectors for gene delivery to the central nervous system

Thomas B. Lentz et al.

NEUROBIOLOGY OF DISEASE (2012)

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Article Neurosciences

Feasibility study for functional test battery of SOD transgenic rat (H46R) and evaluation of edaravone, a free radical scavenger

Masashi Aoki et al.

BRAIN RESEARCH (2011)

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Article Biochemistry & Molecular Biology

Ataxin-2 repeat-length variation and neurodegeneration

Owen A. Ross et al.

HUMAN MOLECULAR GENETICS (2011)

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Article Biochemistry & Molecular Biology

The Dying-Back Phenomenon of Motor Neurons in ALS

Michal Dadon-Nachum et al.

JOURNAL OF MOLECULAR NEUROSCIENCE (2011)

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Article Clinical Neurology

Novel optineurin mutations in patients with familial and sporadic amyotrophic lateral sclerosis

R. Del Bo et al.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2011)

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Article Medicine, General & Internal

Improved Survival with Vemurafenib in Melanoma with BRAF V600E Mutation

Paul B. Chapman et al.

NEW ENGLAND JOURNAL OF MEDICINE (2011)

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Article Clinical Neurology

Dextromethorphan Plus Ultra Low-Dose Quinidine Reduces Pseudobulbar Affect

Erik P. Pioro et al.

ANNALS OF NEUROLOGY (2010)

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Article Neurosciences

Mesenchymal stem cell transplantation in amyotrophic lateral sclerosis: A Phase I clinical trial

L. Mazzini et al.

EXPERIMENTAL NEUROLOGY (2010)

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Review Clinical Neurology

Smoking and the risk of amyotrophic lateral sclerosis: a systematic review and meta-analysis

Alvaro Alonso et al.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2010)

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Article Clinical Neurology

Phase 2 study of sodium phenylbutyrate in ALS

Merit E. Cudkowicz et al.

AMYOTROPHIC LATERAL SCLEROSIS (2009)

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Review Genetics & Heredity

AAV as a Gene Transfer Vector for the Treatment of Neurological Disorders: Novel Treatment Thoughts for ALS

Mark E. Hester et al.

CURRENT GENE THERAPY (2009)

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Review Clinical Neurology

Practice Parameter update: The care of the patient with amyotrophic lateral sclerosis: Multidisciplinary care, symptom management, and cognitive/behavioral impairment (an evidence-based review) Report of the Quality Standards Subcommittee of the American Academy of Neurology

R. G. Miller et al.

NEUROLOGY (2009)

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Review Clinical Neurology

Practice Parameter update: The care of the patient with amyotrophic lateral sclerosis: Drug, nutritional, and respiratory therapies (an evidence-based review) Report of the Quality Standards Subcommittee of the American Academy of Neurology

R. G. Miller et al.

NEUROLOGY (2009)

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Review Clinical Neurology

Electrodiagnostic criteria for diagnosis of ALS

Mamede de Carvalho et al.

CLINICAL NEUROPHYSIOLOGY (2008)

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Article Neurosciences

Treatment with edaravone, initiated at symptom onset, slows motor decline and decreases SOD1 deposition in ALS mice

Hidefumi Ito et al.

EXPERIMENTAL NEUROLOGY (2008)

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Article Clinical Neurology

Analysis of survival and prognostic factors in amyotrophic lateral sclerosis: a population based study

S. Zoccolella et al.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2008)

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Article Clinical Neurology

Sodium phenylbutyrate in Huntington's disease: A dose-finding study

Penelope Hogarth et al.

MOVEMENT DISORDERS (2007)

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Article Clinical Neurology

Investigation of the therapeutic effects of edaravone, a free radical scavenger, on amyotrophic lateral sclerosis (Phase II study)

Hiide Yoshino et al.

AMYOTROPHIC LATERAL SCLEROSIS (2006)

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Review Biochemistry & Molecular Biology

The role of excitotoxicity in the pathogenesis of amyotrophic lateral sclerosis

L. Van den Bosch et al.

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2006)

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Article Medicine, Research & Experimental

Antisense oligonucleotide therapy for neurodegenerative disease

Richard A. Smith et al.

JOURNAL OF CLINICAL INVESTIGATION (2006)

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Article Clinical Neurology

Riluzole therapy for motor neurone disease: An early Australian experience (1996-2002)

MC Zoing et al.

JOURNAL OF CLINICAL NEUROSCIENCE (2006)

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Article Medicine, General & Internal

Trastuzumab after adjuvant chemotherapy in HER2-positive breast cancer

MJ Piccart-Gebhart et al.

NEW ENGLAND JOURNAL OF MEDICINE (2005)

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Article Clinical Neurology

Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis

TM Miller et al.

ANNALS OF NEUROLOGY (2005)

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Article Neurosciences

Amyotrophic lateral sclerosis is a distal axonopathy: evidence in mice and man

LR Fischer et al.

EXPERIMENTAL NEUROLOGY (2004)

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Article Multidisciplinary Sciences

Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model

BK Kaspar et al.

SCIENCE (2003)

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Article Clinical Neurology

An outcome study of riluzole in amyotrophic lateral sclerosis - A population-based study in Ireland, 1996-2000

BJ Traynor et al.

JOURNAL OF NEUROLOGY (2003)

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Article Clinical Neurology

Pathological laughter and crying - A link to cerebellum

J Parvizi et al.

BRAIN (2001)

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Article Clinical Neurology

El Escorial revisited: Revised criteria for the diagnosis of amyotrophic lateral sclerosis

BR Brooks et al.

AMYOTROPHIC LATERAL SCLEROSIS AND OTHER MOTOR NEURON DISORDERS (2000)

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