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A replay of the call will be archived on the site for one week following the call. About VIMIZIM â¢. VIMIZIM (elosulfase alfa) is a treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA).VIMIZIM is the first enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome-a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS).ntil now, patients with Morquio A syndrome didn't have a drug treatment option. This approval provides the community with a therapy and with hope.. Shipments of VIMIZIM to the distribution channels will commence immediately, and BioMarin will begin promotion of VIMIZIM in the U.S. immediately.
Ruxolitinib (trade names Jakafi JAK-É-fye and Jakavi) is a drug for the treatment of intermediate or high-risk myelofibrosis, a type of myeloproliferative disorder that affects the bone marrow, and for polycythemia vera (PCV) when there has been an inadequate response to or intolerance of hydroxyurea.