All Neurological disorders articles
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NewsOrganoid study reveals valproate’s impact on developing brain
German researchers have used cerebral organoids to investigate how the epilepsy medication valproate interferes with early brain development, identifying significant disruption to the extracellular matrix and neuronal maturation that may explain increased neurodevelopmental risks in exposed foetuses.
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ArticleBeyond tangles: why soluble intracellular tau should guide drug discovery
Tau tangles are a hallmark of Alzheimer’s disease and related disorders, but evidence suggests the real damage may come from rare, soluble tau species inside neurons. Targeting these hidden drivers of circuit dysfunction could be key to restoring memory and cognition.
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NewsOutSee wins Longitude Prize award for ALS target discovery
OutSee has secured a Discovery Award from the Longitude Prize on ALS, providing £100,000 in funding and access to genomic data from 9,000 patients. The company will deploy its AI-driven Nomaly platform to identify novel therapeutic targets for amyotrophic lateral sclerosis over a nine-month research programme.
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ArticleWomen in STEM: resilience and leadership in rare disease research
Rare neurological diseases remain one of the most challenging areas in drug discovery, with many patients still lacking treatment options. Dr Nitza Thomasson discusses returning to Servier to lead its rare neurology therapeutic area and explains why resilience, curiosity and persistence are essential for those looking to build a meaningful career in STEM.
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NewsAI-designed viral vectors achieve 50-fold brain enrichment over AAV9
WhiteLab Genomics has presented preclinical data showing that viral vectors designed using artificial intelligence achieved approximately 50-fold higher DNA enrichment in the brain compared to AAV9, with no detectable liver signal following intravenous administration in mice.
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ArticleTurning CRISPR hits into confident drug discovery decisions
Functional genomics is central to modern drug discovery, yet high attrition rates persist. In this article, Dr Salman Tamaddon-Jahromi, a postdoctoral research associate at the University of Cambridge, discusses how end-to-end CRISPR screening strategies, iPSC-derived neuronal models and layered quality control can convert functional genomics signals into actionable therapeutic hypotheses.
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NewsMachine learning identifies five distinct Parkinson’s disease subtypes
A new study from VIB and KU Leuven has revealed that Parkinson’s disease comprises five distinct molecular subtypes, each requiring tailored therapeutic approaches.
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ArticleWhy lower organisms matter for neurodegeneration drug discovery
In the wake of recent government policy aimed at actively replacing animal models in drug discovery, we consider a possible solution to the translational shortfalls of current cellular methodologies for neurodegenerative disease.
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NewsNasal spray reverses age-related brain inflammation in preclinical study
Researchers at Texas A&M University have developed a nasal spray delivering extracellular vesicles that reverses neuroinflammaging in preclinical models. Two doses significantly reduced brain inflammation, restored mitochondrial function and improved memory within weeks, with effects persisting for months. The therapy bypasses the blood-brain barrier and targets inflammatory pathways whilst reactivating cellular energy production.
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ArticleBeyond serendipity: rational design and AI’s expansion of the undruggable target landscape
For decades, drugging the ‘undruggable’ was thought to require luck rather than logic. Today, AI is transforming serendipity into strategy by enabling rational, data-driven approaches to previously inaccessible targets.
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NewsCombining antibodies with natural compounds may improve Alzheimer’s treatment
Researchers at the University of Waterloo have demonstrated that combining anti-amyloid antibodies with naturally derived small molecules, such as resveratrol and curcumin, may improve therapeutic outcomes in Alzheimer’s disease.
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NewsSmall molecule SK-129 targets protein aggregation in Parkinson’s disease
International researchers have identified a small molecule capable of crossing the blood-brain barrier to prevent pathological protein aggregation in Parkinson’s disease, Lewy body dementia and multiple system atrophy.
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NewsMirrored molecules target disordered proteins in Alzheimer’s disease
Researchers at Kobe University have developed a novel approach to Alzheimer’s disease treatment by engineering right-handed amino acid chains that bind to amyloid-beta proteins. The method, inspired by materials science principles of chirality, demonstrated effective inhibition of toxic protein aggregation in mouse brain cell cultures, maintaining cell viability where amyloid-beta alone reduced it by 50 percent.
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ReportCRISPR & Genomics: Turning Data into Confident Drug Discovery Decisions
Early drug discovery has no shortage of genomic data, but confidence remains scarce. This report examines how CRISPR, functional genomics and human-relevant models are being applied to determine which signals matter, how they influence disease biology and which targets and strategies are worth pursuing.
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NewsScientists link IVNS1ABP gene to new ageing disorder
An international research team has identified a previously unknown genetic disease characterised by premature ageing and progressive neurological decline. Using genome sequencing combined with induced pluripotent stem cell technology, scientists traced the condition to a mutation in the IVNS1ABP gene and uncovered disrupted actin dynamics during cell division as the underlying pathological mechanism.
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WebinarWhat it takes to automate high-content imaging at scale
This webinar examines the design trade-offs and technical constraints involved in building a high-throughput robotic imaging pipeline for complex biological workflows.
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NewsCC023 mice reveal how ALS can develop after viral infections
Researchers have discovered a mouse strain that mirrors ALS in humans following a viral infection, offering new insights into how the disease develops, potentially opening new pathways for early diagnosis and drug development.
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NewsMini-stomach organoids grown to improve disease research
Scientists have grown the first multi-regional “mini-stomach” in the lab, creating a new way to study rare genetic stomach diseases and help to develop new treatments for digestive conditions.
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NewsBrain discovery could improve drugs targeting amyloid diseases
New research shows the brain can intentionally form amyloids to store memories, challenging decades of thinking about neurodegenerative disease and pointing towards new strategies for drug discovery.