In a first, a gene therapy seems to slow Huntington disease.
case_series · Level IV
Where this comes from
- Record sourced from PubMed, PMID 41037617.
- Also identified by DOI 10.1126/science.aec7429.
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Abstract
Small study suggests uniQure drug could be first successful treatment for devastating brain disorder.
Medical subject headings
- Genetic Therapy
- Huntingtin Protein
- Huntington Disease