Industry News

Freeline has made the difficult decision to not progress our Fabry disease program further

Orphanet Journal of Rare Diseases recently published an in-depth history of the Sanofi Rare Disease Registries.

It is with great sadness that we learned of the passing of BongKi Lim, Fabry Korea's chairman for the past 22 years. 

Evaluating the direct and indirect costs of Fabry disease 

Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for Pegunigalsidase Alfa for Treatment of Fabry Disease.

Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for Pegunigalsidase Alfa for Treatment of Fabry Disease.

Letter to the Fabry Community from Avrobio

Sangamo Therapeutics Announces Updated Preliminary Phase 1/2 Data Showing Tolerability and Sustained Elevated α-Gal A Enzyme Activity in Patients With Fabry Disease

Freeline announced new data from its ongoing Phase 1/2 MARVEL-1 dose-finding clinical trial of FLT190 for the treatment of Fabry disease and provided updates on its pipeline programs. 

Idorsia Ltd (SIX: IDIA) today announced that after the planned interim analysis of the open-label extension (OLE) of the Phase 3 MODIFY study with lucerastat for the treatment of adult patients with Fabry disease, the study will continue.