When Tomasz Nadolski was seven years old, he began experiencing severe, unexplainable stomach pain. At first, his doctors ...

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 ...

A comprehensive review is shedding new light on Fabry disease, a rare inherited disorder that can progressively damage multiple organs and significantly affect quality of life.

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 ...

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 ...

The rare disease can cause burning, stabbing, or shooting pain in patients. A new report details consensus findings on pain management for patients with Fabry disease. The research was published in ...

(RTTNews) - Chiesi Global Rare Diseases, a unit of the Chiesi Group and Protalix BioTherapeutics, Inc. (PLX) Wednesday said the U.S. Food and Drug Administration (FDA) has approved ELFABRIO ...

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, ...

I’ll start, it has become increasingly recognized these four biomarker in Fabry diseases, and that Plasma Lyso-Gb3 meet the characteristics for that. There is an increasing amount of data that has ...

(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 ...

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 ...