Dr. Grabowski Explains the Effects of Enzyme Therapy

Gregory A. Grabowski, MD, professor, University Cincinnati, Department of Pediatrics, director, Division of Human Genetics, director, Medical Genetics Training Program, Cincinnati Children's Hospital Medical Center, discusses the immense benefit that enzyme therapy provides to patients with Gaucher disease, a debilitating lysosomal storage disease.

The dramatic benefits of enzyme therapy became increasingly evident 5 years after the therapy came into the market. While delivering a talk at a patient conference Grabowski recalls noticing the shift in the appearance of the patients; they no longer carried crutches and looked healthy. The way patients looked before enzyme therapy was gone and would remain gone.

Although the costs are a concern the benefits of the treatment is remarkable. He heralds the Gaucher disease enzyme treatment as amazing with very few problems associated with the treatment.

Grabowski remarks that it is an amazing experience to see patients get better. In the past 20 years he’s witnessed 1200-1300 patients become healthy after receiving the treatment. The comparison of benefits between the enzyme therapy and the supportive therapies used before is remarkable and is a total transformation in healthcare.