Designed to reinforce lucerastat as the first oral therapy suitable for all patients with Fabry disease, irrespective of their mutation type. The program is expected to support a potential regulatory ...
Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~ ...
Idorsia Ltd (SIX: IDIA) announces that the results of lucerastat treatment over 42-months and the kidney biopsy data will be presented at the 22 nd Annual WORLDSymposiumTM, taking place from February ...
~ Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~ ~ Stable Lyso-Gb3 levels maintained post-dosing, regardless of enzyme ...
Total Revenue of $634M, up 17% Year-over-Year at CER Cash Position of $294M, a $44M Increase in 2025 Proposed Acquisition by BioMarin Expected to Close in Q2 2026, Subject to Closing Conditions ...
(RTTNews) - uniQure N.V. (QURE), a gene therapy company developing treatments for severe genetic diseases, announced updated preliminary Phase 1/2a data for AMT-191, its investigational AAV gene ...
UniQure (NASDAQ:QURE) is one of the most promising low-cost stocks to buy now. On February 6, UniQure reported positive updated data from its Phase I/IIa trial of AMT-191, which is a gene therapy for ...
Add Yahoo as a preferred source to see more of our stories on Google. uniQure's AAV gene therapy has displayed early efficacy in Fabry disease UniQure has ceased dosing in two cohorts in a Phase I/IIa ...
SAN DIEGO - Gene therapy company uniQure N.V. (NASDAQ:QURE) announced updated preliminary data from its Phase I/IIa trial of AMT-191, an investigational gene therapy for Fabry disease, showing ...
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