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Targeting gene regulation may hold key to future Alzheimer’s therapies
Researchers have discovered that Alzheimer’s disease is driven by a deeper loss of gene regulation in brain cells – offering potential new targets for future therapies.
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Disease Research
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Scientists spotlight lung disease as fast-track model for ageing drugs
Researchers have identified idiopathic pulmonary fibrosis (IPF) as a powerful model for exploring treatments that target the biology of ageing.
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Animal-free drug discovery is closer with QSP
Quantitative Systems Pharmacology (QSP) is fast becoming a standard tool in drug development, offering a human-relevant way to predict drug effects before the clinic. Dr Josh Apgar of Certara explains how it is helping to cut reliance on animal testing and speed discovery.
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Inside the search-and-develop model tackling 1,000 untreated skin diseases
With over 1,000 skin diseases lacking approved treatments, a search-and-develop model is changing how new therapies are sourced and developed. Chief Scientific Officer, Jacob Pontoppidan Thyssen, outlines the strategy behind it.
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Chronic neuron overactivation drives Parkinson’s cell death
New research from Gladstone Institutes shows that chronic overactivation of dopamine-producing neurons can directly trigger their death, offering new insights into why these cells deteriorate in Parkinson’s disease which could lead to potential therapies to slow its progression.
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Scientists chart ovarian reserve to help advance new infertility treatments
UCLA scientists have created the first detailed map of how the ovarian reserve forms in primates, offering new insights – and potential new treatments – for infertility, polycystic ovary syndrome (PCOS) and hormone-related conditions.
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Inside the Alzheimer’s study backing stem cells, not drugs
In this first-in-human Alzheimer’s study, Wnt-activated autologous stem cells are delivered intracerebroventricularly (directly into the brain) to address neuronal loss, while also reducing amyloid and tau biomarkers and improving cognition. Early data from this regenerative approach could help early drug discovery teams shape target selection, biomarker development and trial design.
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Cholesterol drug restores brain barrier function in 22q11.2 syndrome models
Researchers have found that mitochondrial dysfunction in the blood-brain barrier may drive neuropsychiatric disease in 22q11.2 deletion syndrome – and that a cholesterol drug could restore barrier function and ease symptoms.
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Chikungunya’s 2025 surge sparks urgent research response
The 2025 chikungunya outbreak has surged from the Indian Ocean to Europe, prompting an urgent global research response. With no antivirals and limited vaccine access, laboratories and biotech firms are under pressure to deliver solutions fast.
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Repurposed epilepsy drugs show potential for new autism therapies
Researchers identified hyperactivity in a specific brain region as a driver of autism-like behaviours in mice – and reversed these symptoms using drugs first designed to treat epilepsy.
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New therapy targets triglycerides to prevent aneurysms
A new study shows high triglyceride levels directly cause abdominal aortic aneurysms – highlighting triglyceride-lowering therapies as a potential new treatment.
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Researchers identify early intervention target for cerebral palsy dystonia
Researchers have developed an easy-to-use method for measuring leg dystonia in children with cerebral palsy – allowing doctors to personalise treatments more effectively.
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New CRISPR breakthrough could transform sickle cell treatment
Researchers at UNSW Sydney have unveiled a next-generation CRISPR tool that could lead to safer treatments for genetic disorders, including Sickle Cell.
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FDA-approved asthma drug may protect against food allergy reactions
An existing asthma drug has been shown to almost completely prevent life-threatening allergic reactions to food in mice, after researchers found a previously unknown genetic pathway linked to anaphylaxis.
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Lithium deficiency identified as key Alzheimer’s trigger
New research from Harvard Medical School shows that natural lithium deficiency in the brain may be a key early factor driving Alzheimer’s disease – which allow for the development of new approaches to preventing and reversing cognitive decline.
