Amyotrophic lateral sclerosis: translating genetic discoveries into therapies
Recent advances in sequencing technologies and collaborative efforts have led to
substantial progress in identifying the genetic causes of amyotrophic lateral sclerosis (ALS) …
substantial progress in identifying the genetic causes of amyotrophic lateral sclerosis (ALS) …
The multifaceted role of neurofilament light chain protein in non-primary neurological diseases
The advancing validation and exploitation of CSF and blood neurofilament light chain
protein as a biomarker of neuroaxonal damage has deeply changed the current diagnostic …
protein as a biomarker of neuroaxonal damage has deeply changed the current diagnostic …
[HTML][HTML] Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
TM Miller, ME Cudkowicz, A Genge… - … England Journal of …, 2022 - Mass Medical Soc
Background The intrathecally administered antisense oligonucleotide tofersen reduces
synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with …
synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with …
Current state and future directions in the therapy of ALS
L Tzeplaeff, S Wilfling, MV Requardt, M Herdick - Cells, 2023 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder
affecting upper and lower motor neurons, with death resulting mainly from respiratory failure …
affecting upper and lower motor neurons, with death resulting mainly from respiratory failure …
Tofersen: first approval
HA Blair - Drugs, 2023 - Springer
Tofersen (Qalsody™) is an antisense oligonucleotide being developed by Biogen for the
treatment of amyotrophic lateral sclerosis (ALS). On 25 April 2023, tofersen was approved in …
treatment of amyotrophic lateral sclerosis (ALS). On 25 April 2023, tofersen was approved in …
New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022
JS Jiang, Y Wang, M Deng - Frontiers in pharmacology, 2022 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder that
primarily affects motor neurons in the brain and spinal cord. In the recent past, there have …
primarily affects motor neurons in the brain and spinal cord. In the recent past, there have …
Neurofilament light chain in drug development for amyotrophic lateral sclerosis: a critical appraisal
M Benatar, J Wuu, MR Turner - Brain, 2023 - academic.oup.com
Interest in amyotrophic lateral sclerosis (ALS) biomarkers has grown exponentially over the
course of the last 25 years, with great hope that they might serve as tools to facilitate the …
course of the last 25 years, with great hope that they might serve as tools to facilitate the …
Fluid biomarkers for amyotrophic lateral sclerosis: a review
KE Irwin, U Sheth, PC Wong, TF Gendron - Molecular neurodegeneration, 2024 - Springer
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by
the loss of upper and lower motor neurons. Presently, three FDA-approved drugs are …
the loss of upper and lower motor neurons. Presently, three FDA-approved drugs are …
Gene therapy in amyotrophic lateral sclerosis
Since the discovery of Cu/Zn superoxide dismutase (SOD1) gene mutation, in 1993, as the
first genetic abnormality in amyotrophic lateral sclerosis (ALS), over 50 genes have been …
first genetic abnormality in amyotrophic lateral sclerosis (ALS), over 50 genes have been …
Recent progress of the genetics of amyotrophic lateral sclerosis and challenges of gene therapy
H Wang, LP Guan, M Deng - Frontiers in neuroscience, 2023 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by the
degeneration of motor neurons in the brain and spinal cord. The causes of ALS are not fully …
degeneration of motor neurons in the brain and spinal cord. The causes of ALS are not fully …