Medical Education for Rare Diseases

The Sanofi Rare Diseases Medical Affairs team educates Health Care Professionals in all aspects of the rare diseases we champion. We support the pipeline and commercial products by delivering scientific information and education to Health Care Professionals, researchers, and payers, with the ultimate goal of improved patient care.

This website facilitates educational opportunities for Health Care Professionals through (1) web-based learning, and (2) keeping you informed about Scientific Education Programs conducted in collaboration with the Rare Diseases Medical Science Liaison team. We encourage you to reach out and connect with us to further discuss your educational needs. Please click on this link to contact us.



Fabry Disease


Gaucher Disease




Pompe Disease




How Registries Help Advance Research

For people who are diagnosed with rare disease, they often have a feeling of isolation. They often have a feeling of being the only one who is responsible for explaining their disease to their own physicians.

So what a rare disease registry can provide is that it can provide a global community for each and every one of those patients. Rare disease registries are open structured studies and they can enroll individuals all over the world in order to share their stories and their experiences. It's really powerful for patients and physicians and scientists to be able to bring all of those experiences together and to create shared knowledge. In order to further understanding for the disease community, because registries run for many years, we can understand more about the long-term disease course and we can also understand more about the long-term effects of treatment. This can be extremely impactful for families, for clinicians and for the patient to anticipate and understand how that patient might do over time. A registry is much more than just a database. I think of the registry as the people. So first and foremost the patients, the patients who provide their invaluable data to become that global community. A registry is also the clinicians, the nurses, the genetic counselors, the physicians who are entering these data into the system. The registry is also the people who are asking the important research questions to be able to elucidate the questions that we don't know about a specific disease and to be able to meet those unmet medical needs for the patient community. Rare diseases by their very nature are extremely isolating for both patients and for clinicians. So one of the lasting impacts of the rare disease registries are to create the global community that didn't exist 30 years ago and there's a legacy there that will continue as we move forward and will continue on for years to come.



Medical Information


Grants / Studies


MAT-US-2200282, v1.0
Expires: 3/14/2024