Freeline will be progressing to the second dose cohort in the MARVEL-1 study immediately with first patient dosing expected in mid-2022
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.
Throughout 2020, the COVID-19 pandemic posed many challenges for both FOS and the wider Fabry disease community. The members of the FOS Steering Committee and I would like to thank all patients and caregivers involved in the registry for their extremely valuable contributions and for their continued commitment to [...]
In December 2020 and March 2021, people with Fabry Disease, representing five European countries shared their perspectives on standard questionnaires often used to capture the experiences of people with Fabry Disease and the ideal profile of new tools, designed to more accurately record the Fabry Disease symptoms and impacts. [...]
Amicus Therapeutics Announces European Commission Approval of Galafold® (migalastat) for Adolescents with Fabry DiseaseCharlotte2021-09-16T16:08:20+02:00
Amicus Therapeutics Announces European Commission Approval of Galafold® (migalastat) for Adolescents with Fabry Disease