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.
Publication Name: The New England Journal of Medicine
Subject: Health
ISSN: 0028-4793
Year: 2001
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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.
Publication Name: The New England Journal of Medicine
Subject: Health
ISSN: 0028-4793
Year: 2004
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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.
Publication Name: The New England Journal of Medicine
Subject: Health
ISSN: 0028-4793
Year: 2007
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