نتایج جستجو برای: vi mps

تعداد نتایج: 47894  

Journal: :IEEE Access 2021

In this research, a new technique is developed for reducing the order of high-order continuous interval systems. The model denominator derived using Anderson corollary and Routh table. Numerator by matching formulated Markov parameters (MPs) time moments (TMs). Distinctive features proposed approach are: (i) New simpler expressions MPs TMs; (ii) Retaining not only TMs but also while deriving mo...

2010
Roberto Giugliani Andressa Federhen Maria Verônica Muñoz Rojas Taiane Vieira Osvaldo Artigalás Louise Lapagesse Pinto Ana Cecília Azevedo Angelina Acosta Carmen Bonfim Charles Marques Lourenço Chong Ae Kim Dafne Horovitz Denize Bonfim Denise Norato Diane Marinho Durval Palhares Emerson Santana Santos Erlane Ribeiro Eugênia Valadares Fábio Guarany Gisele Rosone de Lucca Helena Pimentel Isabel Neves de Souza Jordão Correa José Carlos Fraga José Eduardo Goes José Maria Cabral José Simionato Juan Llerena Laura Jardim Liane Giuliani Luiz Carlos Santana da Silva Mara L. Santos Maria Angela Moreira Marcelo Kerstenetzky Márcia Ribeiro Nicole Ruas Patricia Barrios Paulo Aranda Rachel Honjo Raquel Boy Ronaldo Costa Carolina Souza Flavio F. Alcantara Silvio Gilberto A. Avilla Simone Fagondes Ana Maria Martins

Mucopolysaccharidoses (MPS) are rare genetic diseases caused by the deficiency of one of the lysosomal enzymes involved in the glycosaminoglycan (GAG) breakdown pathway. This metabolic block leads to the accumulation of GAG in various organs and tissues of the affected patients, resulting in a multisystemic clinical picture, sometimes including cognitive impairment. Until the beginning of the X...

2014
Violetta Opoka-Winiarska

Introduction One of the most important manifestations of mucopolysaccharidosis (MPS) type I, II and VI is a progressive disease of the osteoarticular system. The evaluation of the disease advancement is difficult due to the complexity of symptoms. The characteristic features are progressive limitation of joint mobility and joint pain. These symptoms affect the quality of patient life. A uniform...

Journal: :PloS one 2016
Calogera M Simonaro Shunji Tomatsu Tracy Sikora Francyne Kubaski Michael Frohbergh Johana M Guevara Raymond Y Wang Moin Vera Jennifer L Kang Lachlan J Smith Edward H Schuchman Mark E Haskins

BACKGROUND We previously demonstrated the therapeutic benefits of pentosan polysulfate (PPS) in a rat model of mucopolysaccharidosis (MPS) type VI. Reduction of inflammation, reduction of glycosaminoglycan (GAG) storage, and improvement in the skeletal phenotype were shown. Herein, we evaluate the long-term safety and therapeutic effects of PPS in a large animal model of a different MPS type, M...

2017
Cristóbal Colón J. Victor Alvarez Cristina Castaño Luís G. Gutierrez-Solana Ana M. Marquez María O’Callaghan Félix Sánchez-Valverde Carmen Yeste María-Luz Couce

The mucopolysaccharidoses (MPSs) are underdiagnosed but they are evaluated in few newborn screening programs, probably due to the many challenges remaining, such as the identification of late-onset phenotypes. Systematic screening at the onset of clinical symptoms could help to early identify patients who may benefit from specific treatments. The aim of this prospective study was to assess a no...

2016
Ali Al Kaissi Jochen Hofstaetter Gerlinde Weigel Franz Grill Rudolf Ganger Susanne Gerit Kircher

INTRODUCTION A 13-year-old child was clinically diagnosed with mucopolysaccharidosis type VI-Maroteaux-Lamy syndrome (MPS VI) at the age of 5 years, and the diagnosis was confirmed biochemically and genetically (homozygous mutation in ARSB gene). At that time, his older brother manifested with increasing severe mental retardation. His urinary glycosaminoglycan excretion in urine was elevated, b...

2016
Gustavo Adolfo Giraldo Paola Ayala-Ramírez Juan Carlos Prieto Reggie García-Robles Johanna Carolina Acosta

INTRODUCTION Maroteaux-Lamy syndrome, or mucopolysaccharidosis (MPS) type VI, is an autosomal recessive lysosomal storage disease caused by a deficient activity of the enzyme arylsulfatase B (ARSB), required to degrade dermatan sulfate. The onset and progression of the disease vary, producing a spectrum of clinical presentation. So far, 133 mutations have been reported. The aim of this study is...

2008
S. Susanne Kircher G. Gert Lubec

Susanne Kircher, Institute of medical Chemistry, Währingerstr. 10, A-1090 Vienna (Austria) Dear Sir, Acid glycosaminoglycans (AGAG) have been implicated in the selective glomerular filtration of proteins, but so far their specific function has remained elusive. In 1978, Kanwar and Farquhar [1] reported the presence of AGAG in the glomerular basement membrane (GBM). Further studies showed that h...

Journal: :Investigative ophthalmology & visual science 1986
L E Stramm R J Desnick M E Haskins G D Aguirre

Feline mucopolysaccharidosis VI (MPS VI) is a recessively inherited lysosomal storage disease resulting from a deficiency of arylsulfatase B (ASB). Previous histopathologic findings have indicated that the disease is expressed morphologically in non-pigmented retinal pigment epithelial cells (RPE) in the posterior pole and superior equatorial regions by the accumulation of vacuolated inclusions...

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