Spinraza is part of the Antisense Therapeutics class and treats Musculoskeletal Conditions. Antisense therapeutics are used to treat certain types of genetic disorders or infections. Antisense therapeutics can work by making a nucleic acid that can "turn off" the particular gene that is causing the disease. They are often not a cure, but can maintain and treat symptoms. Spinraza is only available as a brand name drug. Compare antisense therapeutics.

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FDA Approves Two New Orphan Drugs: Rubraca and Spinraza

Roni Shye - January 26, 2017

Recently, the FDA approved two new orphan drugs, Rubraca and Spinraza.

But first, what is an orphan drug?

An Orphan drug is a medication used to treat a rare disease. By definition, a rare disease is one that affects less than 200,000 patients in the United States, according to the Orphan Drug Act or 1983.

Because some diseases are so rare, a lot of pharmaceutical manufacturers are hesitant to develop medications for the small amount of people that are affected. See More

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