News

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”

As recently reported, Christine Lavery, President of Fabry international Network, sadly passed away on Tuesday 19th December 2017, in hospital, surrounded by her family following a brief illness. Christine was a formidable lady who cared passionately for every MPS and FIN member both past and present. Her efforts saw her work with patients, families and … Continue reading “Important Announcement by the Fabry International Network Board”

It is with the heaviest of hearts that we announce the death of Mrs Christine Lavery MBE, Chief Executive of the Society for Mucopolysaccharide Diseases (MPS Society) Christine sadly passed away on Tuesday 19th December 2017 in hospital surrounded by her family, following a brief illness. Christine has tirelessly championed the MPS Society from its … Continue reading “”