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Intended for US Payers

Rare diseases

Rare diseases R&D

Leading the way to discovery in a connected ecosystem of academic, foundation, and industry research

Our research investigates:

Illustration representing fusion proteins.
Fusion proteins

Illustration representing small molecules.
Small molecules

Illustration representing enzyme replacement therapy.
Enzyme replacement therapy

Search for a specific clinical-stage project

FDA-approved products in rare diseases

Our current portfolio includes approved treatments for:

Acid sphingomyelinase
deficiency (ASMD)

Product information

Fabry disease

Product information

Gaucher disease

Product information

Mucopolysaccharidosis type I (MPS I)

Product information

Pompe disease

Product information

Search for other FDA-approved products

Explore our other areas of focus

Illustration of immune cells with antibodies to represent the therapeutic area of immunology and inflammation.

Immunology and
inflammation

Illustration of the human brain to represent the therapeutic area of neurology.

Neurology

Illustration of a tumor to represent the therapeutic area of oncology.

Oncology

Illustration of a white blood cell, a red blood cell, and platelets to represent the therapeutic area of rare blood disorders.

Rare blood disorders

Let’s connect

Schedule a meeting with a Sanofi Market Access team member to discuss how our therapies can bring value to your plan and members living with rare diseases.

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