Enzyme-Replacement Therapy in Life-Threatening Hypophosphatasia | Zendy

Michael P. Whyte | Zendy; Cheryl R. Greenberg | Zendy; Nada Salman | Zendy; Michael B. Bober | Zendy; William H. McAlister | Zendy; Deborah Wenkert | Zendy; Bradley J. Van Sickle | Zendy; Jill H. Simmons | Zendy; Terence S. Edgar | Zendy; Martin Bauer | Zendy; Mohamed A. Hamdan | Zendy; Nick Bishop | Zendy; Richard E. Lutz | Zendy; Mairead McGinn | Zendy; Stanley Craig | Zendy; Jean N. Moore | Zendy; John W. Taylor | Zendy; Robert Cleveland | Zendy; William R. Cranley | Zendy; Ruth Lim | Zendy; Tom D. Thacher | Zendy; J. Mayhew | Zendy; Matthew Downs | Zendy; José Luís Millán | Zendy; Alison Skrinar | Zendy; Philippe Crine | Zendy; Hal Landy | Zendy

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Enzyme-Replacement Therapy in Life-Threatening Hypophosphatasia

Author(s) -

Michael P. Whyte,

Cheryl R. Greenberg,

Nada Salman,

Michael B. Bober,

William H. McAlister,

Deborah Wenkert,

Bradley J. Van Sickle,

Jill H. Simmons,

Terence S. Edgar,

Martin Bauer,

Mohamed A. Hamdan,

Nick Bishop,

Richard E. Lutz,

Mairead McGinn,

Stanley Craig,

Jean N. Moore,

John W. Taylor,

Robert Cleveland,

William R. Cranley,

Ruth Lim,

Tom D. Thacher,

J. Mayhew,

Matthew Downs,

José Luís Millán,

Alison Skrinar,

Philippe Crine,

Hal Landy

Publication year - 2012

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1106173

Subject(s) - hypophosphatasia , medicine , enzyme replacement therapy , rickets , alkaline phosphatase , gastroenterology , mucopolysaccharidosis , parathyroid hormone , pediatrics , endocrinology , vitamin d and neurology , disease , calcium , biochemistry , chemistry , enzyme

Hypophosphatasia results from mutations in the gene for the tissue-nonspecific isozyme of alkaline phosphatase (TNSALP). Inorganic pyrophosphate accumulates extracellularly, leading to rickets or osteomalacia. Severely affected babies often die from respiratory insufficiency due to progressive chest deformity or have persistent bone disease. There is no approved medical therapy. ENB-0040 is a bone-targeted, recombinant human TNSALP that prevents the manifestations of hypophosphatasia in Tnsalp knockout mice.

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