Pioneering research and treatment of lysosomal storage disorders
Lysosomal storage disorders are a cornerstone of our presence in rare diseases, and the medical area for which we are most well-known. These disorders, which are known as “orphan diseases” include conditions such as: Gaucher disease, Fabry disease, Pompe disease, and Mucopolysaccharidosis 1 (MPS 1), and affect only a few thousand people worldwide.
Sanofi’s breakthrough work in genetic engineering and recombinant DNA manufacturing enabled the large-scale production of enzyme replacement therapies, which work by breaking down the fatty substances or sugars that accumulate in cells due to the lack of native enzyme.
Often diagnosed between the ages 20 and 40, more than 23,000 Australians are living with multiple sclerosis or MS.
MS is a disease that affects each person differently, with symptoms ranging from numbness in the limbs or forgetfulness, to paralysis or loss of vision. It is caused when the body’s immune system attacks the central nervous system, damaging the myelin sheath—the protective layer covering the nerves that carry signals between the brain and spinal cord and the rest of the body.
We are committed to maintaining long-term partnerships with patients, physicians, advocates, and researchers and making a significant impact on people living with MS.