Improving clinical trial outcomes in amyotrophic lateral sclerosis

MC Kiernan, S Vucic, K Talbot, CJ McDermott… - Nature Reviews …, 2021 - nature.com
Individuals who are diagnosed with amyotrophic lateral sclerosis (ALS) today face the same
historically intransigent problem that has existed since the initial description of the disease in …

[HTML][HTML] A comprehensive review of amyotrophic lateral sclerosis

S Zarei, K Carr, L Reiley, K Diaz, O Guerra… - Surgical neurology …, 2015 - ncbi.nlm.nih.gov
Amyotrophic lateral sclerosis (ALS) is a late-onset fatal neurodegenerative disease affecting
motor neurons with an incidence of about 1/100,000. Most ALS cases are sporadic, but 5 …

Cell death

RS Hotchkiss, A Strasser, JE McDunn… - New England Journal …, 2009 - Mass Medical Soc
This review is a primer on the three main mechanisms of cell death: apoptosis, autophagy-
associated cell death, and necrosis. The authors discuss the clinical implications of each of …

The PRO-ACT database: design, initial analyses, and predictive features

N Atassi, J Berry, A Shui, N Zach, A Sherman, E Sinani… - Neurology, 2014 - AAN Enterprises
Objective: To pool data from completed amyotrophic lateral sclerosis (ALS) clinical trials and
create an open-access resource that enables greater understanding of the phenotype and …

FTD and ALS: a tale of two diseases

R Ferrari, D Kapogiannis, ED Huey… - Current Alzheimer …, 2011 - ingentaconnect.com
The first reports of disorders that in terms of cognitive and behavioral symptoms resemble
frontotemporal dementia (FTD) and in terms of motor symptoms resemble amyotrophic …

Heat shock transcription factor 1 as a therapeutic target in neurodegenerative diseases

DW Neef, AM Jaeger, DJ Thiele - Nature reviews Drug discovery, 2011 - nature.com
Neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease,
Huntington's disease, amyotrophic lateral sclerosis and prion-based neurodegeneration are …

Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses

T Kirkegaard, J Gray, DA Priestman… - Science translational …, 2016 - science.org
Lysosomal storage diseases (LSDs) often manifest with severe systemic and central
nervous system (CNS) symptoms. The existing treatment options are limited and have no or …

Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS

M Benatar, J Wuu, PM Andersen, N Atassi, W David… - Neurology, 2018 - AAN Enterprises
Objective To examine the safety and tolerability as well as the preliminary efficacy of
arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in …

Heat shock proteins and autophagy pathways in neuroprotection: from molecular bases to pharmacological interventions

B Penke, F Bogár, T Crul, M Sántha, ME Tóth… - International journal of …, 2018 - mdpi.com
Neurodegenerative diseases (NDDs) such as Alzheimer's disease, Parkinson's disease and
Huntington's disease (HD), amyotrophic lateral sclerosis, and prion diseases are all …

Targeting protein homeostasis in sporadic inclusion body myositis

M Ahmed, PM Machado, A Miller, C Spicer… - Science translational …, 2016 - science.org
Sporadic inclusion body myositis (sIBM) is the commonest severe myopathy in patients more
than 50 years of age. Previous therapeutic trials have targeted the inflammatory features of …