European Experts Outline Organ-Specific Therapeutic Goals for Fabry Disease

A European panel of experts has defined a set of organ-specific therapeutic goals for Fabry disease, based on a systematic literature review and expert consensus. These therapeutic goals are discussed in the study, “European expert consensus statement on therapeutic goals in Fabry disease,” recently published in Molecular…

Early, Multidisciplinary Treatment with ERTs Recommended to Improve Fabry Outcomes

Early therapeutic intervention with enzyme replacement therapies (ERTs), along with a multidisciplinary follow-up, should be initiated at a young age in Fabry patients to promote the best outcomes and prevent disease progression in both adult and pediatric patients. These are the conclusions of the review, “Therapeutic goals…