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Intravenous enzyme replacement therapy: hospital vs home
British Journal of Nursing, 2010Two surveys were carried out to establish the status of enzyme replacement therapy (ERT) for lysosomal storage diseases in Italy. The first was a national survey covering the regional reference centres (RRCs) for these diseases; replies disclosed that 57.7% of patients are on ERT, administered almost exclusively in hospital settings (local hospital 60 ...
Parini, R +4 more
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2004
AbstractThis chapter traces the development of the field of enzyme replacement. It explores the major remaining problem — i.e., treatment of lysosomal storage diseases with a major neurologic component, because of insulation of the brain from the therapeutic enzyme by the blood-brain barrier.
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AbstractThis chapter traces the development of the field of enzyme replacement. It explores the major remaining problem — i.e., treatment of lysosomal storage diseases with a major neurologic component, because of insulation of the brain from the therapeutic enzyme by the blood-brain barrier.
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Enzyme replacement therapy for Gaucher disease
Expert Opinion on Biological Therapy, 2008Gaucher disease is the most common lysosomal storage disease, and the first lysosomal storage disease for which a specific therapy has been developed. Enzyme replacement therapy, with glucocerebrosidase purified from human placentae, was introduced in 1991. Recombinant human glucocerebrosidase, produced by Chinese hamster ovary cells in tissue culture,
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Enzyme Replacement Therapy in a Gaucher Family
Journal of the National Medical Association, 2018Gaucher disease is a lipid storage disorder due to deficiency of beta glucocerebrosidase. It's an autosomal recessive disease and as a result of this enzyme deficiency, glucocerebroside accumulates in various types of tissues like liver, brain spleen and bone marrow.
Neslihan, Erdem +3 more
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2018
Enzyme replacement therapy is a therapeutic approach in which the specific enzyme that is absent or inactive in affected individuals is replaced with a functional enzyme molecule derived from biological sources or produced by biotechnology. A large number and variety of enzyme defects have been identified in humans.
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Enzyme replacement therapy is a therapeutic approach in which the specific enzyme that is absent or inactive in affected individuals is replaced with a functional enzyme molecule derived from biological sources or produced by biotechnology. A large number and variety of enzyme defects have been identified in humans.
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Industrializing enzyme replacement therapy development
Molecular Genetics and Metabolism, 2019Today there are 10 FDA approved enzyme replacement therapy (ERTs), yet there are thousands of rare diseases where ERTs could extend patient lives. The pathway to develop ERTs is well understood. Besides the high costs of GMP manufacturing, we think the development of ERTs is being inhibited at the preclinical stage by the current research paradigm ...
Ana C. Puhl, Sean Ekins
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1983
Publisher Summary This chapter discusses the recent developments in the field of enzyme replacement therapy, describes the successes achieved, and indicates procedures that are expected to improve prospects for enzyme replacement therapy in the future.
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Publisher Summary This chapter discusses the recent developments in the field of enzyme replacement therapy, describes the successes achieved, and indicates procedures that are expected to improve prospects for enzyme replacement therapy in the future.
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Recombinant Enzyme Replacement Therapy in Hypophosphatasia
2015Hypophosphatasia (HPP) is a rare monogenetic and multisystemic disease with involvement of different organs, including bone, muscle, kidney, lung, gastrointestinal tract and the nervous system. The exact metabolic mechanisms of the effects of TNAP deficiency in different tissues are not understood in detail.
Christine, Hofmann +7 more
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Prenatal Enzyme-Replacement Therapy
Obstetric Anesthesia Digest(N Engl J Med. 2022;387(23):2189–2193. doi: 10.1056/NEJMe2211515) Genetic diseases are difficult to treat, and though treatments have been developed none are curative. One still developing treatment is enzyme replacement therapy, and it is currently the standard of care for several lysosomal disorders. A recent study reported the first use of
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Enzyme Replacement Therapy in Feline Mucopolysaccharidosis I
Molecular Genetics and Metabolism, 2001Enzyme replacement therapy (ERT) has long been considered an approach to treating lysosomal storage disorders caused by deficiency of lysosomal enzymes. ERT is currently used to treat Gaucher disease and is being developed for several lysosomal storage disorders now that recombinant sources of the enzymes have become available.
E D, Kakkis +11 more
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