Mucopolysaccharidosis Type I definition

Mucopolysaccharidosis Type I or "MPS I" means a condition in which individuals are missing an enzyme to break down large sugar molecules. This disorder can impact many different organs and tissue leading to developmental delays if not identified and treated early.
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Examples of Mucopolysaccharidosis Type I in a sentence

  • In North Carolina, legislation sets the fees for newborn screening; legislation would be required to add and fund new tests for the panel. Between March of 2015 and June of 2016, three additional conditions were added to the federal RUSP: Pompe (Glycogen Storage Disease Type II), MPS-I (Mucopolysaccharidosis Type I), and X-ALD (X-linked Adrenoleukodystrophy).

  • The Review will assess the clinical benefits achieved through the use of LSDP medicines, ensure the ongoing viability of the program; and ensure testing and access requirements for the medicine remain appropriate.This Review Protocol for Mucopolysaccharidosis Type I (MPS I) medicine was prepared by HealthConsult.

  • Table 8.1 presents the research questions to address ToR 7 and the data sources which will be used to answer each of the research questions.Table 8.1: Research questions to address ToR 7 ToR 7 research questionsAbbreviations: LSDP, life saving drugs program; MPS I, Mucopolysaccharidosis Type I disease; ToR, term of reference Horizon scans are implemented to detect emerging healthcare technologies and innovations and inform stakeholders.

  • Additionally, the processes for the implementation of two LSDs, Pompe and Mucopolysaccharidosis Type I (MPS-1), have already begun and the testing methodology will have to be modified to accommodate the expanded panel.

  • The respondents and participants are all in year 3 of their study.

  • Mucopolysaccharidosis Type I (MPS I) – added to RUSP February 2016 MPS I is a rare genetic disorder caused by a change in a single human gene.

  • Details on the individual data sources are provided in Appendix A.Table 5.1: Research questions to address ToR 4 ToR 4 research questionsAbbreviations: LSDP, life saving drugs program; MPS I, Mucopolysaccharidosis Type I disease; ToR, term of reference The following sections explain how each of the identified data sources will be used to inform the analysis undertaken for each of the research questions.

  • Active, enrolling subjects Pilot Projects:LDN 6710 “Lysosomal Storage Disease: Health, Development, and Functional Outcomes in Preschool Children” (Dr. Michael Msall PI).LDN 6714 “A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis Type I (Drs.

  • Mucopolysaccharidosis Type I (MPS I) is an autosomal recessive disorder that is caused by a deficiency in the enzyme α-L-iduronidase (IDUA).

  • Details on the individual data sources are provided in Appendix A.Table 2.1: Research questions to address ToR 1 ToR 1 research questionsAbbreviations: LSDP, life saving drugs program; MPS I, Mucopolysaccharidosis Type I; ToR, term of reference a Includes pharmaceutical sponsor Note: The MPS I registry was considered to be a potential source and was included in the draft Review Protocol.

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