Rare Diseases

There are more than 7,000 rare diseases, collectively affecting more than 350 million people around the world. Sanofi is committed and focused on caring for the rare diseases community.

JJ, living with Acid Sphingomyelinase deficiency (ASMD), United States

Rare diseases are difficult to diagnose and treat. There are many barriers to a timely diagnosis. Patients and their families often endure a long journey involving specialty doctors, tests, and misdiagnoses. Focusing on these uncommon and underserved medical conditions, Sanofi’s Rare Diseases franchise is committed to offering sustainable, potentially transformative healthcare options and aspires to achieve better care for rare.

Nikita Khadse and Natalia Vargas,  Sanofi Scientists, United States
Nikita Khadse and Natalia Vargas, Sanofi Scientists, United States

With over 40 years working alongside the rare diseases community, Sanofi is grounded in both the science behind these conditions and the experiences of the people who face them. We proudly maintain one of the largest rare diseases pipelines in the industry across multiple disease modalities.

Brian, living with Gaucher disease  Brian is one of the first patients treated for Gaucher disease
Brian, living with Gaucher disease Brian is one of the first patients treated for Gaucher disease

In the United States, we currently have approved products for patients with Fabry disease, Gaucher disease type 1, Mucopolysaccharidosis (MPS) disease type 1, Pompe disease, and Acid sphingomyelinase deficiency (ASMD).

Layla Bral, Sanofi Associate Scientist, Ghent, Belgium
Layla Bral, Sanofi Associate Scientist, Ghent, Belgium

Even when a treatment has been proven tolerated and effective, Sanofi continues to research therapies that will improve the standard of care. This means building on its scientific understanding and striving to develop more therapies with the potential to improve the lives of those living with rare diseases. This includes research in other nephrological, neurologic, metabolic and neuromuscular rare diseases.

We pledge to continue to advance sustainable access to better treatments, deliver the standard of care to improve lives, transform the diagnostic odyssey, and build meaningful connections with our rare diseases community.
 

Inspired by patients worldwide

Hsieh

Living with Fabry disease, Taiwan

Nick

Living with MPS I disease, United States

Andrea

Living with Pompe disease, Denmark

MAT-US-2311475-v1.0-02/2024