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Can UX111 Script History As The First FDA-Approved Treatment For Sanfilippo Syndrome Type A?

By RTTNews Staff Writer   ✉  | Published:  | Google News Follow Us  | Join Us
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Shares of Ultragenyx Pharmaceutical Inc. (RARE) are down 35% from their 52-week high of $60.37, recorded last September, and trade around $39.

This biopharmaceutical company is focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases. It has 4 commercial products - Crysvita for the treatment of X-linked hypophosphatemia, or XLH, and tumor-induced osteomalacia, or TIO; Mepsevii for the treatment of mucopolysaccharidosis VII, or MPSVII or Sly Syndrome; Dojolvi for the treatment of long-chain fatty acid oxidation disorders, or LC-FAOD; and Evkeeza for the treatment of homozygous familial hypercholesterolemia, or HoFH.

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Global Economics Weekly Update -May 18 – May 22, 2026

May 22, 2026 14:46 ET
Minutes of the latest Fed policy session was the highlight of the week along with survey data on the U.S. housing market. In Europe, survey data signaled the trends in the euro area private sector. Further, consumer price inflation data from the U.K. was in focus. In Asia, various economic indicators from China drew attention to the health of the economy.

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