New clinical insight in amyotrophic lateral sclerosis and innovative clinical development from the non-profit repurposing trial of the old drug guanabenz

A Ambrosini, E Dalla Bella, M Ravasi… - Frontiers in …, 2024 - frontiersin.org
Drug repurposing is considered a valid approach to accelerate therapeutic solutions for rare
diseases. However, it is not as widely applied as it could be, due to several barriers that …

Amyotrophic lateral sclerosis: current therapeutic perspectives

V Kumar, T Kashav, MI Hassan - Pathology, prevention and therapeutics …, 2019 - Springer
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease, characterized by
progressive motor neuron degeneration, muscle weakness, paralysis, and ultimately death …

Developing models for cachexia and their implications in drug discovery

M Konishi, N Ebner, S von Haehling… - Expert opinion on …, 2015 - Taylor & Francis
Introduction: Cachexia is a complex metabolic syndrome associated with underlying illness
and characterized by loss of muscle with or without loss of fat mass. Systemic inflammation …

[HTML][HTML] The critical role of membralin in postnatal motor neuron survival and disease

B Yang, M Qu, R Wang, JE Chatterton, XB Liu, B Zhu… - Elife, 2015 - elifesciences.org
Hitherto, membralin has been a protein of unknown function. Here, we show that membralin
mutant mice manifest a severe and early-onset motor neuron disease in an autosomal …

A chemogenomic approach is required for effective treatment of amyotrophic lateral sclerosis

G Pampalakis, G Angelis, E Zingkou… - Clinical and …, 2022 - Wiley Online Library
ALS is a fatal untreatable disease involving degeneration of motor neurons. Μultiple
causative genes encoding proteins with versatile functions have been identified indicating …

CDNF rescues motor neurons in three animal models of ALS by targeting ER stress

F De Lorenzo, P Lüningschrör, J Nam, F Pilotto, E Galli… - BioRxiv, 2020 - biorxiv.org
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease primarily
afflicting motor neurons (MNs) of the spinal cord, brainstem, and motor cortex, leading to …

[HTML][HTML] Endoplasmic reticulum, oxidative stress and their complex crosstalk in neurodegeneration: proteostasis, signaling pathways and molecular chaperones

G Ambrosi, P Milani - AIMS Molecular Science, 2017 - aimspress.com
Cellular stress caused by protein misfolding, aggregation and redox imbalance is typical of
neurodegenerative disorders such as Parkinson's disease (PD) and Amyotrophic Lateral …

Disruption of endoplasmic reticulum proteostasis in age-related nervous system disorders

DB Medinas, Y Hazari, C Hetz - Cellular Biology of the Endoplasmic …, 2021 - Springer
Endoplasmic reticulum (ER) stress is a prominent cellular alteration of diseases impacting
the nervous system that are associated to the accumulation of misfolded and aggregated …

Translating protein phosphatase research into treatments for neurodegenerative diseases

JR Sundaram, ICJ Lee… - Biochemical Society …, 2017 - portlandpress.com
Many of the major neurodegenerative disorders are characterized by the accumulation of
intracellular protein aggregates in neurons and other cells in brain, suggesting that errors in …

Molecular Insights into the Role of ER Stress in Neurodegenerative Diseases

MT Kabir - Current Thoughts on Dementia: From Risk Factors to …, 2022 - Springer
Endoplasmic reticulum (ER) stress can take place due to the disruptions in the structure and
functions of ER, along with the changes in calcium homeostasis and buildup of misfolded …