Hypophosphatasia in adolescents and adults: overview of diagnosis and treatment.


Journal

Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA
ISSN: 1433-2965
Titre abrégé: Osteoporos Int
Pays: England
ID NLM: 9100105

Informations de publication

Date de publication:
Aug 2020
Historique:
received: 22 07 2019
accepted: 11 02 2020
pubmed: 13 3 2020
medline: 18 2 2021
entrez: 13 3 2020
Statut: ppublish

Résumé

This article provides an overview of the current knowledge on hypophosphatasia-a rare genetic disease of very variable presentation and severity-with a special focus on adolescents and adults. It summarizes the available information on the many known mutations of tissue-nonspecific alkaline phosphatase (TNSALP), the epidemiology and clinical presentation of the disease in adolescents and adults, and the essential diagnostic clues. The last section reviews the therapeutic approaches, including recent reports on enzyme replacement therapy (EnzRT).

Identifiants

pubmed: 32162014
doi: 10.1007/s00198-020-05345-9
pii: 10.1007/s00198-020-05345-9
doi:

Substances chimiques

Alkaline Phosphatase EC 3.1.3.1

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

1445-1460

Auteurs

M L Bianchi (ML)

Laboratorio sperimentale di ricerche sul metabolismo osseo infantile, Centro Malattie Metaboliche Ossee, Istituto Auxologico Italiano IRCCS, Milan, Italy. ml.bianchi@auxologico.it.

N J Bishop (NJ)

Department of Oncology and Metabolism, University of Sheffield, Sheffield Children's NHS Foundation Trust, Sheffield, UK.

N Guañabens (N)

Servicio de Reumatología, Hospital Clínic, Universidad de Barcelona, IDIBAPS CIBERehd, Barcelona, Spain.

C Hofmann (C)

Children's Hospital, University of Würzburg, Würzburg, Germany.

F Jakob (F)

Orthopedic Centre for Musculoskeletal Research, University of Würzburg, Würzburg, Germany.

C Roux (C)

INSERM U1153 APHP Centre, Université de Paris, Department of Rheumatology, Paris, France.

M C Zillikens (MC)

Department of Internal Medicine, Endocrinology Section, Erasmus Medical Centre, Rotterdam, The Netherlands.

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Classifications MeSH