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First Brain-Delivered Gene Therapy Approved for AADC Deficiency

Source link : https://www.newshealth.biz/health-news/first-brain-delivered-gene-therapy-approved-for-aadc-deficiency/

The FDA granted accelerated approval to eladocagene exuparvovec (Kebilidi) to treat children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency, the agency announced Thursday. The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have decreased AADC activity that causes severe disability, including gross motor […]

Author : News Health

Publish date : 2024-11-14 21:55:52

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