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One-time gene therapy could “transform” Huntington’s treatment landscape
uniQure’s positive topline data “are the most convincing in the field to date” and indicate the gene therapy’s potential disease-modifying effects.
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Huntington disease
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Novartis to advance novel Huntington’s disease therapy
As part of the agreement with Novartis Pharmaceuticals, PTC will receive an initial $1 billion to develop the potential first-in-class oral therapy.
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New breakthrough can effectively deliver medications into the brain
Scientists have found a way to effectively transport medication into the brain which could lead to improved treatments for neurological and neurodegenerative diseases.
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New drug for Huntington lowers levels of disease-causing protein
A new drug for Huntington disease is found to be safe and lowers levels of the abnormal protein that causes the debilitating disease in patients.
