Sly syn·drome

an autosomal recessive disorder due to a deficiency of a β-glucuronidase; defective lysosomal degradation of dermatan sulfate, heparan sulfate, and chondroitin sulfate; cellular function disrupted in most tissues.

Synonym(s): mucopolysaccharidosis type VII (1)

Sly syn·drome

Synonym(s): mucopolysaccharidosis type VII (1)

Farlex Partner Medical Dictionary © Farlex 2012

Sly syndrome

(slī)

A type of mucopolysaccharidosis (MPS VII) characterized by the presence of dermatan sulfate, heparan sulfate, and chondroitin sulfate in the urine, corneal clouding, enlargement of the liver and spleen, skeletal abnormalities, and sometimes intellectual disability.

Sly,

William S., U.S. pediatrician, 1932–.

Sly syndrome - beta-glucuronidase deficiency that causes short stature with hepatosplenomegaly and frequent pulmonary infections; may also cause slow development, coarse facies, and clouded corneas. Synonym(s): type VII mucopolysaccharidosis

Medical Eponyms © Farlex 2012

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References in periodicals archive

Food and Drug Administration (FDA) has approved MEPSEVII[TM] (vestronidase alfa), the first medicine approved for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome).

INDICATION: MEPSEVII is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome).

ULTRAGENYX ANNOUNCES FDA APPROVAL OF MEPSEVII[TM]: Mepsevii (Vestronidase Alfa) is the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis Vii

The US Food and Drug Administration (FDA) has granted approval to Ultragenyx Pharmaceutical's enzyme replacement therapy, Mepsevii (vestronidase alfa), intended for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in both children and adults, it was reported yesterday.

US Food and Drug Administration grants approval to Ultragenyx's Mepsevii

Steiner et al., "Clinical course of sly syndrome (mucopolysaccharidosis type VII)," Journal of Medical Genetics, vol.

First Report on Fetal Cerebral Polyglucosan Bodies in Mucopolysaccharidosis Type VII

Another compound, UX003 for MPS 7 (Sly Syndrome), is expected to begin Phase 1/2 clinical trials later this year.

ULTRAGENYX GAINS WORLDWIDE RIGHTS FOR TRIHEPTANOIN

Snyder of the Harvard Medical School in Boston and his colleagues recently implanted neural progenitor cells from the brains of healthy newborn mice into newborn mice that have Sly syndrome, a rare and fatal metabolic disorder (SN: 8/10/91, p.93).

Breathing time for liquid crystal states

M2 EQUITYBITES-November 16, 2017-Ultragenyx passes US FDA's approval for Mepsevii for the rare genetic enzyme disorder Sly syndrome

Ultragenyx passes US FDA's approval for Mepsevii for the rare genetic enzyme disorder Sly syndrome

M2 PHARMA-November 16, 2017-Ultragenyx passes US FDA's approval for Mepsevii for the rare genetic enzyme disorder Sly syndrome

Ultragenyx passes US FDA's approval for Mepsevii for the rare genetic enzyme disorder Sly syndrome