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 ...

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 ...

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...

uniQure N.V. QURE on Friday announced updated preliminary safety and exploratory efficacy data from 11 patients in its Phase ...

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

Scientists identify a neuronal enzyme linked to amyloid plaque formation, opening new possibilities for targeted Alzheimer’s disease treatment.

Xanthine oxidase inhibitors, primarily allopurinol and febuxostat, are the mainstay of treatment. The goal of chronic gout management includes lowering serum urate levels to below 6 mg/dL. 5 Overall, ...

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need ...

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...

In the United States about 5%-8% of the population is affected by an autoimmune disorder. Other reports, however, estimate a higher prevalence of various types of autoimmune disorders due to complex ...

Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 Deficiency First Pivotal Data Readout in Children Expected ...

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...