Enzyme-Replacement Therapy in Life-Threatening Hypophosphatasia
Enzyme-Replacement Therapy in Life-Threatening Hypophosphatasia
About this item
Full title
Author / Creator
Whyte, Michael P , Greenberg, Cheryl R , Salman, Nada J , Bober, Michael B , McAlister, William H , Wenkert, Deborah , Van Sickle, Bradley J , Simmons, Jill H , Edgar, Terence S , Bauer, Martin L , Hamdan, Mohamed A , Bishop, Nick , Lutz, Richard E , McGinn, Mairead , Craig, Stanley , Moore, Jean N , Taylor, John W , Cleveland, Robert H , Cranley, William R , Lim, Ruth , Thacher, Tom D , Mayhew, Jill E , Downs, Matthew , Millán, José Luis , Skrinar, Alison M , Crine, Philippe and Landy, Hal
Publisher
Waltham, MA: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
Waltham, MA: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
In this study of perinatal and infantile hypophosphatasia, patients received ENB-0040, a bone-targeted, recombinant, human tissue-nonspecific isozyme of alkaline phosphatase that is lacking in this disease. Rickets healed, and developmental milestones and pulmonary function improved.
Hypophosphatasia is the inborn error of metabolism that is cha...
Alternative Titles
Full title
Enzyme-Replacement Therapy in Life-Threatening Hypophosphatasia
Authors, Artists and Contributors
Author / Creator
Greenberg, Cheryl R
Salman, Nada J
Bober, Michael B
McAlister, William H
Wenkert, Deborah
Van Sickle, Bradley J
Simmons, Jill H
Edgar, Terence S
Bauer, Martin L
Hamdan, Mohamed A
Bishop, Nick
Lutz, Richard E
McGinn, Mairead
Craig, Stanley
Moore, Jean N
Taylor, John W
Cleveland, Robert H
Cranley, William R
Lim, Ruth
Thacher, Tom D
Mayhew, Jill E
Downs, Matthew
Millán, José Luis
Skrinar, Alison M
Crine, Philippe
Landy, Hal
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_journals_927566797
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_927566797
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1106173
