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Tags: rare | enzyme | disorder | drug | ultragenyx | fda

FDA Approves Drug for Rare Enzyme Disorder

FDA Approves Drug for Rare Enzyme Disorder
(Copyright DPC)

Wednesday, 15 November 2017 01:16 PM EST

The U.S. Food and Drug Administration approved on Wednesday a new treatment made by Ultragenyx Pharmaceutical Inc. for a rare genetic disorder that can dramatically reduce life expectancy.

The drug is designed to treat patients with mucopolysaccharidosis type VII, or MPS VII, a condition caused by an enzyme deficiency that affects tissue and organs, including the heart, and stunts growth.

The disorder affects fewer than 150 patients worldwide.

The drug, Mepsevii, was given to 23 patients who were treated for up to 164 weeks, the FDA said. After 24 weeks, patients taking the drug were able to walk on average 18 meters further within six minutes than those who did not receive the treatment.

© 2022 Thomson/Reuters. All rights reserved.


FDA-Approved-Drugs
Federal regulators have approved a new treatment made by Ultragenyx Pharmaceutical Inc. for a rare genetic disorder that can dramatically reduce life expectancy.The drug is designed to treat patients with mucopolysaccharidosis type VII.
rare, enzyme, disorder, drug, ultragenyx, fda
114
2017-16-15
Wednesday, 15 November 2017 01:16 PM
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