News

If so, Fulcrum Research Group, an independent, third-party market research firm, is seeking Fabry patients or their caregivers for a market research study. Specifically, the research will aim to gain a better understanding of individual patients’ experiences and opinions about: Their journey from initial symptoms to present day (through the diagnostic process, experiences with healthcare … Continue reading “Are you interested in sharing your experience with Fabry Disease?”

The latest newsletter is now available and includes a word from the new FIN President, Lut De Baere, as well as a round up of the 6th FIN Fabry Expert meeting in Lithuania, latest news from the pharma industry and information from Japan Fabry Disease Patients and Family Association. Read here

Idorsia Pharmaceuticals, a Swiss biotech company, is developing lucerastat, a new oral therapy for patients with Fabry Disease. The Phase 3 clinical study MODIFY has just enrolled its first patients and will investigate the use of lucerastat monotherapy for the treatment of adult patients with Fabry disease, irrespective of their genetic mutation type. The MODIFY … Continue reading “Lucerastat, a new oral therapy for Fabry patients in development”

The March Newsletter available now.  Find out how you can help raise funds for FIN with AVROBIO.  Latest news from MetabERN and see our President Jack Johnson receive the 2018 PAL award at WORLD Symposium 2018 Read here  

Fast Track designation highlights high unmet medical need in the treatment of Fabry disease Protalix BioTherapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to pegunigalsidase alfa, or PRX-102, the Company’s plant cell-expressed recombinant, pegylated, cross-linked α-galactosidase-A candidate for the treatment of Fabry disease. The FDA’s Fast Track designation … Continue reading “Protalix BioTherapeutics’ pegunigalsidase alfa Receives Fast Track Designation from the U.S. Food and Drug Administration”