0000000000267673

AUTHOR

Irene Tramacere

showing 3 related works from this author

The MITOS system predicts long-term survival in amyotrophic lateral sclerosis

2015

ObjectiveThe choice of adequate proxy for long-term survival, the ultimate outcome in randomised clinical trials (RCT) assessing disease-modifying treatments for amyotrophic lateral sclerosis (ALS), is a key issue. The intrinsic limitations of the ALS Functional Rating Scale-Revised (ALSFRS-R), including non-linearity, multidimensionality and floor-effect, have emerged and its usefulness argued. The ALS Milano-Torino staging (ALS-MITOS) system was proposed as a novel tool to measure the progression of ALS and overcome these limitations. This study was performed to validate the ALS-MITOS as a 6-month proxy of survival in 200 ALS patients followed up to 18 months.MethodsAnalyses were performe…

MalePredictive Value of TestWalkingLogistic regressionALS; MOTOR NEURON DISEASE; NEUROMUSCULAR; RANDOMISED TRIALS; Adult; Aged; Amyotrophic Lateral Sclerosis; Communication; Deglutition; Disability Evaluation; Disease Progression; Double-Blind Method; Female; Humans; Male; Middle Aged; Noninvasive Ventilation; Predictive Value of Tests; ROC Curve; Respiration; Self Care; Survival Analysis; Walking; Neurology (clinical); Psychiatry and Mental Health; Surgery; Arts and Humanities (miscellaneous); Medicine (all)law.inventionALS long-term survival ALSFRS-RDisability EvaluationRandomized controlled triallawNEUROMUSCULARAmyotrophic lateral sclerosisMOTOR NEURON DISEASEALS; MOTOR NEURON DISEASE; NEUROMUSCULAR; RANDOMISED TRIALS; Neurology (clinical); Psychiatry and Mental Health; Surgery; Arts and Humanities (miscellaneous)CommunicationRespirationMedicine (all)Area under the curveMiddle Agedals motor neuron disease neuromuscular randomised trialsPsychiatry and Mental HealthPredictive value of testsDisease ProgressionSettore MED/26 - NeurologiaFemaleSurvival AnalysiHumanAdultmedicine.medical_specialtyNOSwallowingDouble-Blind MethodArts and Humanities (miscellaneous)Predictive Value of TestsInternal medicinemedicineRANDOMISED TRIALSHumansSurvival analysisAgedNoninvasive VentilationReceiver operating characteristicbusiness.industryAmyotrophic Lateral Sclerosisals; motor neuron disease; neuromuscular; randomised trials; adult; aged; amyotrophic lateral sclerosis; communication; deglutition; disability evaluation; disease progression; double-blind method; female; humans; male; middle aged; noninvasive ventilation; predictive value of tests; roc curve; respiration; self care; survival analysis; walking; neurology clinical; psychiatry and mental health; surgery; arts and humanities ; medicinemedicine.diseaseSurvival AnalysisSurgeryDeglutitionSelf CareALS; MOTOR NEURON DISEASE; NEUROMUSCULAR; RANDOMISED TRIALS; Adult; Aged; Amyotrophic Lateral Sclerosis; Communication; Deglutition; Disability Evaluation; Disease Progression; Double-Blind Method; Female; Humans; Male; Middle Aged; Noninvasive Ventilation; Predictive Value of Tests; ROC Curve; Respiration; Self Care; Survival Analysis; Walking; Surgery; Arts and Humanities (miscellaneous); Neurology (clinical); Psychiatry and Mental HealthROC CurveSurgeryNeurology (clinical)ALSbusinessAmyotrophic Lateral Sclerosi
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Protein misfolding, amyotrophic lateral sclerosis and guanabenz: Protocol for a phase II RCT with futility design (ProMISe trial)

2017

IntroductionRecent studies suggest that endoplasmic reticulum stress may play a critical role in the pathogenesis of amyotrophic lateral sclerosis (ALS) through an altered regulation of the proteostasis, the cellular pathway-balancing protein synthesis and degradation. A key mechanism is thought to be the dephosphorylation of eIF2α, a factor involved in the initiation of protein translation. Guanabenz is an alpha-2-adrenergic receptor agonist safely used in past to treat mild hypertension and is now an orphan drug. A pharmacological action recently discovered is its ability to modulate the synthesis of proteins by the activation of translational factors preventing misfolded protein accumula…

0301 basic medicineOncologyPathologyamyotrophic lateral sclerosisamyotrophic lateral sclerosis; motor neurone disease; neuromuscular disease; randomized clinical trial guanabenz; unfolded protein response; adrenergic alpha-2 receptor agonist s; age of onset; amyotrophic lateral sclerosis; disease progression; double-blind method; endoplasmic reticulum stress; guanabenz; humans; italy; medical futility; neuroprotective agents; proteostasis deficienciesamyotrophic lateral sclerosis; motor neurone disease; neuromuscular disease; randomized clinical trial guanabenz; unfolded protein response; Medicine (all)randomized clinical trial guanabenzHelsinki declaration0302 clinical medicineProtocolAdrenergic alpha-2 Receptor Agonists1506Amyotrophic lateral sclerosisAge of OnsetGuanabenzMedicine (all)amyotrophic lateral sclerosis; motor neurone disease; neuromuscular disease; randomized clinical trial guanabenz; unfolded protein responseNeurodegenerationamyotrophic lateral sclerosis; motor neurone disease; neuromuscular disease; randomized clinical trial guanabenz; unfolded protein response;amyotrophic lateral sclerosis; guanabenz; motor neurone disease; neuromuscular disease; randomized clinical trial; unfolded protein response; Adrenergic alpha-2 Receptor Agonists; Age of Onset; Amyotrophic Lateral Sclerosis; Disease Progression; Double-Blind Method; Endoplasmic Reticulum Stress; Guanabenz; Humans; Italy; Medical Futility; Neuroprotective Agents; Proteostasis DeficienciesGeneral Medicineunfolded protein responseEndoplasmic Reticulum StressRiluzoleNeuroprotective AgentsNeurologyTolerabilityItalyDisease Progression1713GuanabenzMedical Futilitymedicine.drugmedicine.medical_specialtyamyotrophic lateral sclerosis; motor neurone disease; neuromuscular disease; randomized clinical trial guanabenz; unfolded protein response; Adrenergic alpha-2 Receptor Agonists; Age of Onset; Amyotrophic Lateral Sclerosis; Disease Progression; Double-Blind Method; Endoplasmic Reticulum Stress; Guanabenz; Humans; Italy; Medical Futility; Neuroprotective Agents; Proteostasis Deficiencies; Medicine (all)Neuroprotection03 medical and health sciencesmotor neurone diseaseDouble-Blind MethodInternal medicinemedicineHumansProteostasis Deficienciesbusiness.industryAmbientaleneuromuscular diseaserandomized clinical trialmedicine.diseaseClinical trial030104 developmental biologybusiness030217 neurology & neurosurgery
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Dataset related to article "The unfolded protein response in amyotrophic later sclerosis: results of a phase 2 trial"

2021

The data support the findings of the study "The Unfolded Protein Response in Amyotrophic Later Sclerosis: Results of a Phase 2 Trial". They include demographic and clinical data at baseline, and changes measured by the primary and secondary outcomes at 6 months

GuanabenzAmyotrophic Lateral SclerosisUnfolded Protein Responsephase 2 trial
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