Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation
Amyotrophic lateral sclerosis (ALS) is a devastating disease caused by degeneration of
motor neurons. As with all major neurodegenerative disorders, development of disease …
motor neurons. As with all major neurodegenerative disorders, development of disease …
Frontotemporal lobar degeneration
Frontotemporal lobar degeneration (FTLD) is one of the most common causes of early-onset
dementia and presents with early social–emotional–behavioural and/or language changes …
dementia and presents with early social–emotional–behavioural and/or language changes …
Improving clinical trial outcomes in amyotrophic lateral sclerosis
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 …
historically intransigent problem that has existed since the initial description of the disease in …
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
Expansions of a G4C2 repeat in the C9ORF72 gene are the most common genetic cause of
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two devastating …
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two devastating …
C9orf72-mediated ALS and FTD: multiple pathways to disease
R Balendra, AM Isaacs - Nature Reviews Neurology, 2018 - nature.com
The discovery that repeat expansions in the C9orf72 gene are a frequent cause of
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) has revolutionized …
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) has revolutionized …
An update on genetic frontotemporal dementia
CV Greaves, JD Rohrer - Journal of neurology, 2019 - Springer
Frontotemporal dementia (FTD) is a highly heritable group of neurodegenerative disorders,
with around 30% of patients having a strong family history. The majority of that heritability is …
with around 30% of patients having a strong family history. The majority of that heritability is …
Novel genes associated with amyotrophic lateral sclerosis: diagnostic and clinical implications
Background The disease course of amyotrophic lateral sclerosis (ALS) is rapid and,
because its pathophysiology is unclear, few effective treatments are available. Genetic …
because its pathophysiology is unclear, few effective treatments are available. Genetic …
Genetics of amyotrophic lateral sclerosis: seeking therapeutic targets in the era of gene therapy
N Suzuki, A Nishiyama, H Warita, M Aoki - Journal of human genetics, 2023 - nature.com
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 …
leading to mortality. The main locus of ALS is motor neurons. The success of antisense …
Amyotrophic lateral sclerosis: an update for 2018
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting motor neurons
and other neuronal cells, leading to severe disability and eventually death from ventilatory …
and other neuronal cells, leading to severe disability and eventually death from ventilatory …
ALS genes in the genomic era and their implications for FTD
Amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disease, characterized
genetically by a disproportionately large contribution of rare genetic variation. Driven by …
genetically by a disproportionately large contribution of rare genetic variation. Driven by …