The small molecule successfully targeted the C9orf72 gene that causes amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
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Amyotrophic Lateral Sclerosis (ALS)
Stealth BioTherapeutics reported positive data from a pre-clinical study evaluating SBT-272 in a murine model of Parkinson's disease.
Researchers have uncovered a signalling pathway associated with glutamatergic synapse degeneration in the brains of mice with Alzheimer’s.
Researchers have created a new method to quantify protein droplets involved in neurodegenerative diseases, enhancing the study of treatments.
Scientists demonstrated how to reverse the incorrect localisation of three RNA-binding proteins in ALS, potentially leading to treatments.
Eran Blacher has won the NOSTER & Science Microbiome Prize for discovering the link between the microbiome and neurodegenerative diseases.
How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy
In this article, Dr Jim Burns discusses promising pre-clinical results of how a new platform could treat the root cause of many devastating genetic diseases including myotonic dystrophy type 1.
Researchers have designed an antibody that attaches to MuSK, which prevented early lethality of mice with congenital myasthenia.
Mark Mortenson, CSO of Clene Nanomedicine, discusses the use of proprietary gold nanocrystals as catalysts to prevent the progression of, and act as a treatment for, neurodegenerative disease.
Using X-ray crystallography and cryo-electron microscopy, researchers have elucidated the structure of the SARM1 protein, a target for neurodegeneration.
Researchers report that reduced TDP-43 expression disrupts axonal transport of messenger RNAs to cause neurodegeneration in amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD).
Scientists have shown that a Selenium-based drug-molecule called ebselen and other novel compounds can delay ALS onset in mouse models.
Researchers have found the TBK1 enzyme regulates the degradation and clearance of the huntingtin protein, making it a drug target for Huntington's disease.
The researchers revealed the mechanism by which signalling becomes dysfunctional in upper motor neuron (UMN) diseases, such as amyotrophic lateral sclerosis (ALS).