The treatment of Gaucher disease is poised to enter a new era as a result of advances in the understanding of the cellular pathways at work in the disorder.
Rogerio Vivaldi, MD, has vivid memories of the first patient with Gaucher disease ever treated in Brazil with enzyme replacement therapy.
The mood of the ICHG/ASHG 2011 congress often seemed buoyant as researchers discussed the doors that technology is helping to open.