Truncated version of the SMN2 gene could revolutionise drug discovery
A condensed version of the SMN2 gene could improve discovery of potential therapies for spinal muscular atrophy and other conditions.
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A condensed version of the SMN2 gene could improve discovery of potential therapies for spinal muscular atrophy and other conditions.
Researchers discovered that NPTX2 protein accumulated in cells containing abnormal TDP-43 in FTD and ALS patients.
Researchers have found that using protein-like polymers to inhibit the Keap1/Nrf2 PPI is a powerful therapeutic strategy.
A 2D neuromuscular junction model enables high-throughput screening to discover new treatments for neuromuscular diseases.
Scientists find a way to slow the progression of Huntington’s disease by analysing DNA and find the basis of a potential treatment.
A growing body of evidence pinpoints neuroinflammation as a pivotal factor driving brain-related pathogenesis. Yet, a crucial question lingers: among the various immune cell groups residing within the brain, which one orchestrates this inflammatory reaction?
In the sections that follow, we dive in on the latest research describing the underlying causes of ALS and a closely related condition known as frontotemporal dementia (FTD), two forms of neurodegenerative disease that share striking similarities in their molecular pathologies.
In this interview with Drug Target Review’s Izzy Wood and Dr Isaac Klein, Chief Scientific Officer at Dewpoint Therapeutics, discuss the innovative potential of condensate biology in revolutionising drug discovery. By targeting disease-driving condensates, Dewpoint’s research pushes boundaries by offering new hope for tackling diseases like ALS and colorectal cancer.
According to Swedish study, muscle fibres found in extraocular muscles seem to be resistant and even increase in proportion in those with ALS. This offers new treatment avenues for slowing down the disease.
In this article, Dr Vincent Blomen, Senior Director of Target Discovery at Scenic Biotech, takes us through the realm of rare diseases. In the case of most of these diseases, a singular genetic anomaly often reigns supreme, yet its impact on patients can be vastly divergent. Enter modifier genes, the…
US study identifies promising new target, in protein remnants from an ancient virus, for treating underlying cause of ALS.
The researchers found restoring mitochondrial homeostasis in the diseased neurons could protect the optic nerve cells from being damaged from glaucoma.
Swedish researchers have discovered that by measuring immune cells in the cerebrospinal fluid when diagnosing ALS, it is possible to predict how fast the disease may progress.
US scientists have developed a potential medication for the genetic cause of ALS and dementia, that eliminates the mutated segments of RNA.
Following FDA clearance, Cellenkos will initiate Phase I and Phase Ib trials of CK0803, allogeneic regulatory T cells, in patients with ALS.