MPS III


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MPS III

The American Heritage® Medical Dictionary Copyright © 2007, 2004 by Houghton Mifflin Company. Published by Houghton Mifflin Company. All rights reserved.
References in periodicals archive ?
Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Overview 9
The report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
MPS III, or Sanfilippo syndrome type A, B, C, and D, and MPS IV A, or Morquio syndrome type A, can present with Hurler-like symptoms, particularly in the early-onset variants.
And while physicians worldwide have diagnosed only 25 to 30 people with MPS VII, one in 70,000 individuals in Israel is born with MPS II, while MPS III affects one in 24,000 in the Netherlands.
The product is the firm's first-in-human, adeno-associated viral-based gene therapy for MPS III, which involves a one-time intravenous delivery of a normal copy of the Naglu gene to cells of the central nervous system and peripheral organs with the aim of reversing the effects of the genetic errors that cause the disease.
Global Markets Direct's, 'Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2015', provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)'s therapeutic pipeline.
[USPRwire, Thu Jul 18 2013] Global Markets Direct's, 'Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2013', provides an overview of the indication's therapeutic pipeline.
[ClickPress, Tue Jan 13 2015] Global Markets Direct's, 'Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2014', provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)'s therapeutic pipeline.