All Rare & Genetic Disorders articles – Page 3
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NewsNew UCD centre targets the biggest bottleneck in rare research
The world’s first translational research center dedicated to urea cycle disorders (UCDs) has been launched in Zürich, marking a significant moment in rare disease innovation.
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NewsNasal spray gene therapy shows potential to treat lung diseases
Researchers at Mass General Brigham have developed a new gene therapy delivery system, AAV.CPP.16, that targets the lungs via nasal spray, which could offer a potential breakthrough in respiratory gene therapy.
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ArticleAI at the forefront of age-related disease treatment
The body undergoes changes with age that can lead to conditions like sarcopenia and osteoarthritis, burdening individuals and healthcare systems. Find out how Rejuvenate Biomed uses AI to decode ageing biology and develop combination therapies targeting the root causes of age-related diseases, offering hope for better treatments and quality of ...
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ArticleCracking the code to scalable cell and gene therapy
Cell and gene therapy is rapidly transforming the treatment of complex diseases, yet scaling production efficiently remains a challenge. Dr Tia Harmon from PHC Corporation of North America explores the innovations that are optimising cell expansion to enhance the discovery of advanced therapies.
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NewsRepurposed anti-inflammatory drug may help treat alcohol use disorders
A new study by Scripps Research reveals that apremilast, an FDA-approved anti-inflammatory drug, may help reduce both alcohol consumption and pain sensitivity, offering a potential dual therapy for alcohol use disorder.
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NewsTargeting zombie cells: a new treatment for chronic back pain
McGill University researchers have found that two drugs can eliminate senescent “zombie cells” in spinal discs, offering a potential new treatment for chronic low back pain.
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NewsNew drug combination shows potential to treat enteroviruses
Researchers in Norway have identified a promising oral drug combination that may stop the replication of enteroviruses, a group of viruses responsible for illnesses ranging from the common cold to type 1 diabetes.
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ArticleDriving diversity and leadership in the biotech industry
Judy Ashworth, CMO of Novadip Biosciences, shares her journey in advancing treatments for rare paediatric conditions and championing diversity in STEM.
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NewsNew study identifies key immune cells that prevent food allergies
A specialised group of immune cells in the gut has been found to prevent allergic reactions - a discovery that could lead to new treatments for allergies and autoimmune diseases.
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ArticlePioneering cellular therapies for age-related diseases
Discover how Immorta Bio's cellular therapies are addressing cancer and liver failure, with the potential to transform healthcare.
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NewsStem cell study uncovers shared genes for hearing and vision repair
New research, led by USC Stem Cell scientists, has identified key genetic barriers to sensory cell regeneration in the ear and eye, paving the way for future drug therapies to restore hearing and vision.
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Alzheimer’s treatment targets root causes, not just symptoms
Porosome Therapeutics has made a groundbreaking discovery in Alzheimer’s research by targeting the disease’s molecular causes, focusing on restoring secretory and metabolic functions. This approach could not only slow progression but potentially reverse early-stage pathology.
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ArticleGene silencing: a step forward for rare disease therapy
Gene silencing offers a promising approach to treating rare neurological diseases like H-ABC. With clinical trials on the horizon, find out how targeted therapies could bring real hope to patients and families.
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NewsNew research offers hope for high-grade glioma treatment
High-grade glioma, an aggressive brain cancer affecting both paediatric and adult patients, remains difficult to treat. A collaborative study from the University of Michigan, Dana-Farber Cancer Institute, and the Medical University of Vienna reveals a promising new treatment approach.
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ArticleNew insights into surfactant spread for lung drug delivery
A recent study led by Dr Richard McNair highlights the role of the Marangoni effect in surfactant spreading, offering potential improvements in drug development and delivery for lung diseases.
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ArticleThe future of cell therapy: scaling production for global reach
Scaling up cell therapy is no easy feat. PluriCDMO's Andy Lewin reveals the critical factors for success and how they are helping companies bring life-saving therapies to market.
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NewsRare diseases, big advances: how research is powering a new era of treatments
This Friday, February 28, marks Rare Disease Day 2025, a global initiative raising awareness for the 300 million people living with rare conditions. Dr David Reynolds, CEO of LoQus23 Therapeutics, explores how advancements in rare disease research are driving the development of new treatments.
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NewsBlood pressure drug shows promise in ADHD symptom reduction
A study by the University of Surrey suggests that amlodipine, a common blood pressure medication, could be repurposed as a safer, more accessible treatment for ADHD. This offers a promising alternative to current medications and highlights the potential of drug repurposing in advancing treatment options.
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NewsStudy identifies new genetic targets for neuropsychiatric diseases
Scientists identify thousands of novel enhancers linked to neuronal differentiation and neuropsychiatric disorders, offering new pathways for drug discovery and potential therapeutic targets.
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NewsThe immune cells that fuel food allergies
A new study reveals how specific immune cells contribute to the exaggerated immune response in food allergies, offering hope for improved treatments in the future.