Results From Clinical Trial of Iptacopan in C3G

Carla Nester, MD, University of Iowa Stead Family Children’s Hospital, discusses results from the clinical trial of iptacopan in complement 3 glomerulopathy (C3G).     C3G is a rare kidney disease characterized by damage to kidney glomeruli due to abnormal...

Phase 3 BRIGHT Study in Patients With Fabry Disease

Jack Johnson, Co-Founder and Executive Director of the Fabry Disease Support and Information Group, and VP of the Fabry International Network, discusses the phase 3 BRIGHT study testing Elfabrio (pegunigalsidase alfa-iwx) in patients with Fabry disease.    ...

AAV Gene Therapy in Patients With Gaucher Disease

Reena Sharma, MD, Salford Royal Hospital and University of Manchester, UK, discusses clinical data from a clinical trial testing an AAV gene therapy in patients with Gaucher disease.   Clarification (around 8:52): *malignancies Gaucher disease refers to a group...