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Huntington’s disease: danger of repeated DNA sequences
Scientists find a way to slow the progression of Huntington’s disease by analysing DNA and find the basis of a potential treatment.
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Amyotrophic Lateral Sclerosis (ALS)
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Brain macrophage in Parkinson’s disease neuroinflammation
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?
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A Molecular Portrait of ALS and FTD
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.
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Condensate biology: advancing drug discovery for complex diseases
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.
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Extraocular muscles may hold key to defensive mechanisms against ALS
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.
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The genetic modifier approach: identifying the right target for rare diseases
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…
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Ancient virus holds connections to ALS in people
US study identifies promising new target, in protein remnants from an ancient virus, for treating underlying cause of ALS.
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Scientists discover therapeutic target that could treat glaucoma
The researchers found restoring mitochondrial homeostasis in the diseased neurons could protect the optic nerve cells from being damaged from glaucoma.
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Immune cells in ALS patients: predicting disease progression
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.
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Genetic cause of ALS and dementia repaired by RNA-targeting strategy
US scientists have developed a potential medication for the genetic cause of ALS and dementia, that eliminates the mutated segments of RNA.
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FDA clears Cellenkos IND application for potential ALS treatment
Following FDA clearance, Cellenkos will initiate Phase I and Phase Ib trials of CK0803, allogeneic regulatory T cells, in patients with ALS.
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Protein-based therapy shows promise for individuals with ALS
In a petri dish under an environmental condition reminiscent of ALS, the team found that the protein activates a unique pathway inside cells that increases survival and protects endothelial cells from toxic substances in the blood.
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Gene therapy approach shows promise in treating ALS
In rodent models of ALS, the introduced neuroprotective protein slowed disease progression and increased life span.
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Driving precision neurology forwards with iPSCs and new assays
Precision medicine in neurology is limited by a dearth of clinically relevant models. However, here Dr Evangelos Kiskinis, Assistant Professor at Northwestern University Feinberg School of Medicine, discusses how new technologies such as bioelectronic assays enable real-time, long-term analysis of neurological diseases in a dish, offering a pathway towards identifying…
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Molecular pathway shared by two neurodegenerative disorders found
Scientists have gained deeper knowledge about the mislocalisation of a protein, providing a possible therapeutic target that could have implications in treating dementia.
