Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...
Administration in the mid-to-high dose cohorts of the gene therapy trial will pause pending further evaluation.
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 ...
Chiesi’s abstract presentations will focus on clinical insights and patient-reported outcomes in Fabry disease and ...
Sanofi will take venglustat to regulators for Gaucher disease but an application for Fabry disease is less clear after the failure of a Phase III trial.
Sanofi reported phase 3 success for experimental drug venglustat in treating neurological symptoms of type 3 Gaucher disease ...
For Jack Johnson, getting diagnosed with Fabry disease at age 7 was more of a formality. He’d been experiencing symptoms for several years already, and had watched multiple family members struggle ...
Fabry disease is a rare genetic condition that belongs to a group of disorders referred to as lysosomal storage disease. This condition is also called Anderson-Fabry disease, Fabry’s disease, ...
Fabry disease is a progressive condition that can come with a complex range of symptoms and complications that includes neuropathic pain, skin manifestations, and gastrointestinal issues. If left ...
People with Fabry disease don’t produce enough of the lysosomal enzyme alpha-galactosidase A (alpha-Gal A). Without enough of this enzyme, sphingolipids, a fat-like substance, build up in blood ...
This year’s most-read articles on Fabry disease touched on advancements in therapy, health risks of the disease, and keys to improved diagnosis. The top 5 most-read Fabry disease articles of 2021 on ...
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