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Related to hurler: Hurley, Hurler syndrome
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The company is developing innovative therapies for the treatment of lysosomal storage disorders including neurological symptoms such as Hurler syndrome (MPS I), Hunter syndrome (MPS II), metachromatic leukodystrophy, Sanfilippo A and B syndromes, as well as other diseases with severe CNS manifestations.
* The report reviews pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) by companies and universities/research institutes based on information derived from company and industry-specific sources
Hurler Syndrome occurs when the body lacks a particular enzyme that breaks down glycosaminoglycans (GAGs), a type of sugar molecule utilized by our cells to help build bones, cartilages, tendons, corneas of the eyes, skin and other types of connective tissues.
"These stem cells, modified so they produce large quantities of the enzyme that people with Hurler's syndrome lack, offer great hope for a potential new therapy.
* Coverage of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline on the basis of route of administration and molecule type.
The lysosomes in the cells of children with Hurler syndrome do not have a vital enzyme called IDUA (/a-/L-idunronidase), which causes their cells to accumulate too much of a class of biochemical known as mucopolysaccharides, in this instance dermatan sulfate and heparin sulfate.
With an independent league featuring eight teams, the city of more than 600,000 people attracts hurlers the world over.
If both parents carry a mutation in the gene that underlies Hurler's syndrome, a child has a 1-in-4 chance of getting two defective gene copies.
This report provides comprehensive information on the therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases.
Hurler's syndrome is a lysosomal storage disorder that affects approximately one of every 100,000 children.
This report provides comprehensive information on the therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases.
The article by investigators at the University of Minnesota and scientists from Athersys describes preclinical study results suggesting that the stem cells could provide benefit to patients suffering from lysosomal storage disorders, such as Hurler's Syndrome.