About Fabry Disease

Recent Posts

• FDA grants orphan designation to new Fabry cell therapy GT-GLA-S03
• Sangamo seeks accelerated US approval of gene therapy for Fabry
• Common Fabry symptoms often mimic IBS in adults
• Two of my sons share what it’s like having three siblings with Fabry
• Idorsia outlines new Phase 3 program for lucerastat in Fabry disease
• Jeff’s Journey With Fabry Disease
• Eye vessel abnormalities may signal heart disease in Fabry patients
• We need more oral Fabry disease treatment options that reduce pain
• AMT-191 shows promise, but safety concerns prompt dosing pause
• Guest Voice: Believe us when we say we’re having a bad day