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PEG-ADA

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PEG-ADA

An orphan drug that is useful in treating SCID related to ADA deficiency.
Gale Encyclopedia of Medicine. Copyright 2008 The Gale Group, Inc. All rights reserved.
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Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID).
During enzyme replacement therapy with PEG-ADA, ADA activity ranged from undetectable to 14 555 nmol x [s.sup.-1] x [(L plasma).sup.-1].
Except in those few cases where bone marrow from an HLA/MLR-compatible sibling donor has been available, therapy for ADA deficiency has been based on enzyme replacement with PEG-modified ADA and, since 1990, on gene therapy transfer, the latter requiring concomitant PEG-ADA therapy until recently (24).
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