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MDS 2020

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Promising results from gene therapy trial in advanced PD reported

Specialists have reported promising initial findings from a trial of VY‐AADC01, an adeno-associated virus serotype 2 (AAV2) gene therapy encoding human aromatic L‐amino acid decarboxylase (AADC), in patients with advanced Parkinson’s disease.

Dr Chad Christine from the University of California San Francisco, USA, presented preliminary 3-year data from the PD-1101 trial of the gene therapy.

In the trial, 15 patients were given bilateral, intraoperative MRI-guided putaminal infusions of the gene therapy at three doses (5 patients in each dose cohort). Dr Christine reported that:

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