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Enzyme-replacement therapy in mucopolysaccharidosis I

Article Abstract:

Enzyme replacement therapy may be beneficial for patients with mucopolysaccharidosis I, according to a study of 10 patients. This disease is caused by a deficiency of the enzyme alpha-L-iduronidase. This leads to the accumulation of metabolic byproducts that can damage the liver, heart, joints, and other organs.

Author: Phillips, Jeffrey, Kakkis, Emil D., Muenzer, Joseph, Tiller, George E., Waber, Lewis, Belmont, John, Passage, Merry, Izykowski, Barbara, Doroshow, Robin, Walot, Irv, Hoft, Richard, Neufeld, Elizabeth F.
Publisher: Massachusetts Medical Society
Publication Name: The New England Journal of Medicine
Subject: Health
ISSN: 0028-4793
Year: 2001
Health aspects, Enzymes, Enzyme therapy

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Advances in the treatment of mucopolysaccharidosis type 1

Article Abstract:

Mucopolysaccharidoses are a group of lysosomal storage diseases caused by a deficiency of enzymes that degrade glycosaminoglycans. Characteristics and treatment of this severe disease is discussed.

Author: Muenzer, Joseph, Fisher, Amy
Publisher: Massachusetts Medical Society
Publication Name: The New England Journal of Medicine
Subject: Health
ISSN: 0028-4793
Year: 2004
Development and progression, Connective tissue diseases

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Pluripotency redux - advances in stem-cell research

Article Abstract:

The article states pluripotency as one of the major advances in stem-cell research, which will be of use for reprogramming of adult human cells to produce patient-specific therapies.

Author: Gearhart, John, Pashos, Evanthia E., Prasad, Megana K.
Publisher: Massachusetts Medical Society
Publication Name: The New England Journal of Medicine
Subject: Health
ISSN: 0028-4793
Year: 2007
Science & research, Research, Human cell culture, Stem cell research

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Subjects list: Care and treatment, Mucopolysaccharidosis, Mucopolysaccharidoses, United States
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