Tag #Uniqure

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dw.com

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Sep 26, 01:14

Huntington's Disease Progression Slowed by 75% in Clinical Trial

British researchers announced a 75% reduction in Huntington's disease progression over 36 months in a clinical trial of 29 patients using a new gene therapy, AMT-130, developed with UniQure.

Huntington's Disease Progression Slowed by 75% in Clinical Trial

British researchers announced a 75% reduction in Huntington's disease progression over 36 months in a clinical trial of 29 patients using a new gene therapy, AMT-130, developed with UniQure.

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What are the next steps and potential long-term implications of this breakthrough?

UniQure plans to seek accelerated FDA approval in early 2026, followed by UK and European applications. Successful approval would represent a major advance in treating this currently incurable disease, potentially improving patients' quality of life significantly.

edition.cnn.com

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Sep 25, 10:19

UniQure's Gene Therapy Shows Promise in Slowing Huntington's Disease Progression

A Phase 1/2 study by uniQure showed a 75% reduction in Huntington's disease progression in patients receiving a high dose of AMT-130 gene therapy after 36 months, marking a potential breakthrough in treating this currently incurable condition.

UniQure's Gene Therapy Shows Promise in Slowing Huntington's Disease Progression

A Phase 1/2 study by uniQure showed a 75% reduction in Huntington's disease progression in patients receiving a high dose of AMT-130 gene therapy after 36 months, marking a potential breakthrough in treating this currently incurable condition.

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What is the primary impact of uniQure's Phase 1/2 study results on Huntington's disease treatment?

The study's findings demonstrate a 75% reduction in Huntington's disease progression in patients given a high dose of AMT-130, representing a significant step towards the first disease-modifying treatment. This could fundamentally change the treatment landscape, moving beyond symptom management to directly slowing disease progression.

cnnespanol.cnn.com

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Sep 25, 07:18

Experimental Gene Therapy Shows Significant Slowdown in Huntington's Disease Progression

A Phase 1/2 study by uniQure showed that a high dose of their gene therapy, AMT-130, slowed Huntington's disease progression by 75% in patients after 36 months, marking a potential breakthrough for the currently incurable disease.

Experimental Gene Therapy Shows Significant Slowdown in Huntington's Disease Progression

A Phase 1/2 study by uniQure showed that a high dose of their gene therapy, AMT-130, slowed Huntington's disease progression by 75% in patients after 36 months, marking a potential breakthrough for the currently incurable disease.

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What are the next steps and potential future implications of this research?

uniQure plans to submit data to the FDA in Q1 2026, with potential market launch by the end of 2027 if approved. While further research is necessary, this success could accelerate the development of similar one-time, precision gene therapies for other neurological diseases. The reduction in neurofilament light protein levels further supports the positive effects of this treatment.

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