نتایج جستجو برای: maroteaux lamy syndrome

تعداد نتایج: 622119  

Journal: :Journal of pediatric rehabilitation medicine 2010
Arline McDonald Robert Steiner Kerry Kuehl Sean Turbeville

Increased functional capacity of major organ systems improves the quality of life and contributes to reductions in the morbidity associated with chronic debilitating diseases. Routine endurance tests can be used to gauge the progression of disease and the impact of therapeutic modalities in disorders with multiple organ system involvement such as with Mucopolysaccharidosis type VI (MPS VI). MPS...

2008
DAVID A. BALDWIN Graham Allison Charles W. Kegley

Graham Allison and Gregory F. Treverton, eds. Rethinking America's Security: Beyond Cold War to New World Order. New York: W. W. Norton, 1992,479 pp. John Lewis Gaddis. The United States and the End of the Cold War: Implications, Reconsiderations, Provocations. New York: Oxford University Press, 1992, 301 pp. Michael J. Hogan, ed. The End of the Cold War: Its Meaning and Implications. New York:...

2016
S. Somanadhan P. J. Larkin

BACKGROUND Many rare diseases of childhood are life-threatening and chronically debilitating, so living with a rare disease is an on-going challenge for patients and their families. MPS is one of a range of rare inherited metabolic disorders (IMDs) that come under category 3 of life-limiting conditions, where there is no curative treatment available at present. Although the study of rare diseas...

Journal: : 2022

Kistler, kaynak aldıkları epitel dokusuna göre farklı özellik gösterebilen merkezî konumlu bir lümene ve bağ doku duvarına sahip patolojik boşluklardır. Çocuklarda nadir görülmekle birlikte, erişkinlere daha hızlı büyüme ilerleme özelliği gösterebilmektedirler. Dişlerin gömülü kalmasına sürememesine, mine malformasyonlarına, çene fraktürlerine, dokusunun metaplazi göstermesiyle kanser gelişimin...

Journal: :Journal of pediatric rehabilitation medicine 2010
Celeste Decker Zi-Fan Yu Roberto Giugliani Ida Vanessa D Schwartz Nathalie Guffon Elisa Leão Teles M Clara Sá Miranda J Edmond Wraith Michael Beck Laila Arash Maurizio Scarpa David Ketteridge John J Hopwood Barbara Plecko Robert Steiner Chester B Whitley Paige Kaplan Stuart J Swiedler Susan Conrad Paul Harmatz

BACKGROUND AND METHODS: Growth failure is characteristic of untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to and for up to 240 weeks of weekly infusions of recombinant human arylsulfatase B (rhASB) at 1 mg/kg during Phase 1/2, Phase 2, Phase 3 or Phase 3 Extension clinical trials. Height, weig...

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