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https://sciencedaily.com/releases/2025/01/250116133442.htm
Study finds surprising way that genetic mutation causes Huntington's disease, transforming...
Scientists have discovered a surprising mechanism by which the inherited genetic mutation known to cause Huntington's disease leads to the death of brain...
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https://www.neophore.com/
NeoPhore | Broadening cancer patient access to powerful disease-transforming immunotherapy
Therapeutic approach transiently blocks DNA MMR to mimic known robust and durable clinical responses to immunotherapy of patients with genetically...
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https://www.broadinstitute.org/news/study-finds-surprising-way-genetic-mutation-causes-huntingtons-disease-transforming
Study finds surprising way that genetic mutation causes Huntington’s disease, transforming...
Researchers studying brain cells from Huntington’s patients show that the mutation, which changes over decades, becomes toxic only later in life.
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https://www.southampton.ac.uk/research/highlights/transforming-chronic-respiratory-disease-care
Transforming chronic respiratory disease care | University of Southampton
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https://www.takeda.com/science/areas-of-focus/rare-diseases/
Transforming Rare Disease Care | Takeda Pharmaceuticals
Learn about our commitment to empower rare disease communities. We focus on innovative therapies and comprehensive support to improve patient outcomes and...
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https://www.scientificamerican.com/article/engineered-viruses-are-transforming-neuroscience-and-treating-brain-disease/
Engineered Viruses Are Transforming Neuroscience and Treating Brain Disease | Scientific American
Jun 2, 2025 - Neuroscientists can now make precise genetic tweaks to the neurons that are most affected by brain diseases such as Parkinson’s, Huntington’s and ALS
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https://www.ocutx.com/
Ocular Therapeutix | Transforming Retinal Disease Treatment with AXPAXLI™
Feb 4, 2026 - Discover Ocular Therapeutix's retinal disease treatments. Learn more about AXPAXLI, our potential treatment for wet AMD our sustained-release hydrogel therapy,...
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