Foothills Medical Centre

Breakthrough Gene Therapy Eliminates Need for Enzyme Replacement in Fabry Disease Patients

- Canadian researchers have successfully developed a one-time gene therapy treatment for Fabry disease that restored enzyme production to near-normal levels in all five trial participants within one week. - The world-first trial, published in Nature Communications, used a lentivirus to deliver functional GLA genes to patients' own blood stem cells, allowing three participants to discontinue biweekly enzyme replacement therapy. - Led by Dr. Aneal Khan at Foothills Medical Centre, this pioneering approach represents a potential paradigm shift in Fabry disease management, though long-term monitoring will continue for at least five years.