Literature DB >> 18328977

Achondroplasia.

Geneviève Baujat1, Laurence Legeai-Mallet, Georges Finidori, Valérie Cormier-Daire, Martine Le Merrer.   

Abstract

Achondroplasia (MIM 100800) is the most common non-lethal skeletal dysplasia. Its incidence is between one in 10,000 and one in 30,000. The phenotype is characterized by rhizomelic disproportionate short stature, enlarged head, midface hypoplasia, short hands and lordotic lumbar spine, associated with normal cognitive development. This autosomal-dominant disorder is caused by a gain-of-function mutation in the gene encoding the type 3 receptor for fibroblast growth factor (FGFR3); in more than 95% of cases, the mutation is G380R. The diagnosis is suspected on physical examination and confirmed by different age-related radiological features. Anticipatory and management care by a multidisciplinary team will prevent and treat complications, including cervical cord compression, conductive hearing loss and thoracolumbar gibbosity. Weight counselling, psychosocial guidance and professional integration programmes play an important role in the global quality of life of these patients and their families.

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Year:  2008        PMID: 18328977     DOI: 10.1016/j.berh.2007.12.008

Source DB:  PubMed          Journal:  Best Pract Res Clin Rheumatol        ISSN: 1521-6942            Impact factor:   4.098


  33 in total

Review 1.  Sixteen years and counting: the current understanding of fibroblast growth factor receptor 3 (FGFR3) signaling in skeletal dysplasias.

Authors:  Silvie Foldynova-Trantirkova; William R Wilcox; Pavel Krejci
Journal:  Hum Mutat       Date:  2011-11-16       Impact factor: 4.878

Review 2.  Achondroplasia: Development, pathogenesis, and therapy.

Authors:  David M Ornitz; Laurence Legeai-Mallet
Journal:  Dev Dyn       Date:  2017-03-02       Impact factor: 3.780

Review 3.  Advances in research on and diagnosis and treatment of achondroplasia in China.

Authors:  Yao Wang; Zeying Liu; Zhenxing Liu; Heng Zhao; Xiaoyan Zhou; Yazhou Cui; Jinxiang Han
Journal:  Intractable Rare Dis Res       Date:  2013-05

4.  Pediatric spondylolysis/spinal stenosis and disc herniation: national trends in decompression and discectomy surgery evaluated through the Kids' Inpatient Database.

Authors:  Tridu R Huynh; Carlito Lagman; Fadi Sweiss; Faris Shweikeh; Miriam Nuño; Doniel Drazin
Journal:  Childs Nerv Syst       Date:  2017-06-22       Impact factor: 1.475

Review 5.  Disease-specific complications and multidisciplinary interventions in achondroplasia.

Authors:  Hiroshi Kitoh; Masaki Matsushita; Kenichi Mishima; Yasunari Kamiya; Kenta Sawamura
Journal:  J Bone Miner Metab       Date:  2022-01-14       Impact factor: 2.626

Review 6.  Syndromic Hydrocephalus.

Authors:  Kaamya Varagur; Sai Anusha Sanka; Jennifer M Strahle
Journal:  Neurosurg Clin N Am       Date:  2022-01       Impact factor: 2.509

7.  A registry of achondroplasia: a 6-year experience from the Czechia and Slovak Republic.

Authors:  Martin Pesl; Hana Verescakova; Linda Skutkova; Jana Strenkova; Pavel Krejci
Journal:  Orphanet J Rare Dis       Date:  2022-06-16       Impact factor: 4.303

Review 8.  Achondroplasia: a comprehensive clinical review.

Authors:  Richard M Pauli
Journal:  Orphanet J Rare Dis       Date:  2019-01-03       Impact factor: 4.123

9.  Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model.

Authors:  Davide Komla-Ebri; Emilie Dambroise; Ina Kramer; Catherine Benoist-Lasselin; Nabil Kaci; Cindy Le Gall; Ludovic Martin; Patricia Busca; Florent Barbault; Diana Graus-Porta; Arnold Munnich; Michaela Kneissel; Federico Di Rocco; Martin Biosse-Duplan; Laurence Legeai-Mallet
Journal:  J Clin Invest       Date:  2016-04-11       Impact factor: 14.808

10.  Art in Science: Velázquez and Dwarfism-The Art of Observation.

Authors:  Gary E Friedlaender; Linda K Friedlaender
Journal:  Clin Orthop Relat Res       Date:  2020-01       Impact factor: 4.755

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