Protalix BioTherapeutics and Chiesi Farmaceutici have endured a rejection from the FDA and conducted multiple readouts of a phase 3 trial of their Fabry disease candidate. After many stops and starts, ...

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 ...

NEWARK, Calif.--(BUSINESS WIRE)--AceLink Therapeutics, Inc., a clinical stage biopharmaceutical company developing the next generation of oral substrate reduction therapies (SRTs) to address ...

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 ...

(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 ...

NEWARK, Calif.--(BUSINESS WIRE)--AceLink Therapeutics, Inc., a clinical-stage biopharmaceutical company developing the next generation of oral substrate reduction therapies (SRTs), announced today ...

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.

Protalix BioTherapeutics (PLX) said on Friday that an expert panel of the European Medicines Agency (EMA), endorsed a new dosing regimen for Elfabrio, an enzyme replacement therapy developed by the ...

It’s now known that women, once thought to merely carry the genetic mutation that causes this disorder, can develop symptoms, although they may not be as severe as for men. Fabry disease (or ...