With our leadership in diseases that affect small numbers of patients comes a responsibility to
reach all patients. Because there is little awareness of these conditions, we work to educate
physicians and target patient populations, together with establishing patient registries and
centers of excellence. We believe that effective treatment demands diagnostics as well as therapeutics,
so we have funded the development of improved newborn screening tests for the medical community. Because
these diseases need to be managed closely to ensure compliance and optimal outcomes, we are working with
leading physicians on a disease management approach, by which they monitor each patient's progress
according to an established set of therapeutic goals. This approach is especially important because of
the wide variability in each patient's symptoms and severity of disease. Genzyme continues to innovate,
perhaps even to replace our own successful treatments with newer options such as oral therapies and
ultimately gene therapy. Since drugs for very rare diseases are expensive to develop and cannot attain
the volume to reduce costs, we will continue to work with governments and insurers to demonstrate their
value to patients and society.