Amyotrophic lateral sclerosis (ALS) is an intractable disease that causes respiratory failure
leading to mortality. The main locus of ALS is motor neurons. The success of antisense …

Induced pluripotent stem cells (iPSCs) are increasingly used in the study of disease
mechanisms and the development of effective disease-modifying therapies for …

Summary iPSC-based drug discovery led to a phase 1/2a trial of ropinirole in ALS. 20
participants with sporadic ALS received ropinirole or placebo for 24 weeks in the double …

Simulation is a key tool in population genetics for both methods development and empirical
research, but producing simulations that recapitulate the main features of genomic datasets …

RASopathies are a group of developmental disorders caused by mutations in genes that
regulate the RAS/MAPK pathway and include Noonan syndrome (NS), Costello syndrome …

Background The characteristic structure of motor neurons (MNs), particularly of the long
axons, becomes damaged in the early stages of amyotrophic lateral sclerosis (ALS) …

Amyotrophic lateral sclerosis (ALS) is a progressive, fatal neurodegenerative disease
characterized by selective impairment of upper and lower motor neurons. We aimed to …

Amyotrophic lateral sclerosis (ALS) is an intractable adult-onset neurodegenerative disease
that leads to the loss of upper and lower motor neurons (MNs). The long axons of MNs …

Introduction Amyotrophic lateral sclerosis (ALS) is a progressive and severe
neurodegenerative disease caused by motor neuron death. There have as yet been no …

Mutations within Superoxide dismutase 1 (SOD1) cause amyotrophic lateral sclerosis (ALS),
accounting for approximately 20% of familial cases. The pathological feature is a loss of …