India, Feb. 3 -- uniQure N.V. (QURE), Monday, announced the completion of enrollment of participants in the first cohort of its Phase I/IIa trial of AMT-191 for the treatment of Fabry disease.
Additionally, the Independent Data Monitoring Committee has recommended the company proceed with enrollment in the second cohort of the trial, which is set to begin in the first quarter of 2025.
Fabry disease is a genetic condition resulting from a deficiency of the enzyme alpha-galactosidase A (GLA).
In the Phase I/IIa trial of AMT-191 trial, the company will be evaluating the safety, tolerability, and early signs of efficacy by measuring the expression of lysosomal enzyme aGLA-A. The two dosing cohorts will enrol six adult male patients each who ...