UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...
uniQure N.V. (NASDAQ:QURE) on Friday released initial safety and exploratory efficacy data from the first cohort of its Phase 1/2a trial of AMT-191, an investigational gene therapy for Fabry disease.
STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) ...
Sangamo Therapeutics Inc. (NASDAQ:SGMO) is one of the best penny stocks under $1 to buy now. On June 24, Sangamo Therapeutics announced positive topline results from its registrational Phase 1/2 STAAR ...
CEO Alexander Macrae highlighted continued advancement of the clinical and preclinical pipeline, with a focus on managing cash resources. Macrae stated, "In September, we presented promising detailed ...
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