Last Updated: March 28, 2013
Items marked with a * have been filed for regulatory review.
The platform or technologic approach upon which a treatment is based; either chemically synthesized like a small molecule or polymer drug, or biologic like a protein, antibody, or cell therapy.
Pre-Clinical testing conducted in the lab and on animals to investigate a treatment’s ability to fight against a targeted disease and to demonstrate safety.
Clinical trials on small number of human subjects, usually healthy, primarily to assess safety and potential side effects at different dosages.
Clinical trials on affected patients, primarily to test efficacy; usually randomized and placebo-controlled.
Clinical trials on affected patients to further test efficacy, benefits, and potential side effects; usually randomized, placebo-controlled, and blinded.
Focusing on uncommon and underserved medical conditions, our Rare Disease business unit develops breakthrough therapies for patients who might otherwise have few treatment options. Read more
Fabrazyme® (agalsidase beta)
Fabry disease, label expansion
Niemann-Pick disease type B
AAV-hAADC gene therapy
AAV-sFLT gene therapy
Age-related macular degeneration
Pompe disease, 2nd generation enzyme
Fabry disease, substrate reduction
We are committed to becoming a longterm partner to the MS community by working to deliver scientific advancements that will have a significant impact on the unmet needs of people living with MS.Read more
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