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 ...
Researchers from Drexel University’s College of Arts and Sciences and College of Medicine have found a potential new therapeutic target for Alzheimer’s disease. Expanding on their previous research on ...
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 ...
A protein that has long frustrated diabetes researchers is suddenly at the center of a very different fight. By targeting an enzyme called protein tyrosine phosphatase 1B, or PTP1B, scientists are ...
Researchers present novel therapeutics that rejuvenate a previously undruggable mitochondrial regulator of aging through a new mode of enzyme activation. Leveraging a newly identified biophysical ...
AMT-191 has been granted both Orphan Drug and Fast Track designation by the U.S. Food and Drug Administration.