Literature DB >> 30868735

CHRNG-related nonlethal multiple pterygium syndrome: Muscle imaging pattern and clinical, histopathological, and molecular genetic findings.

Laura Carrera-García1,2, Daniel Natera-de Benito1,2, Klaus Dieterich3, Marta G G de la Banda4, Adrien Felter5, Emili Inarejos6, Anna Codina1,2, Cristina Jou1,2,7, Monica Roldan8, Francesc Palau9,10,11,12, Janet Hoenicka10,13, Jordi Pijuan10, Carlos Ortez1,2, Jessica Expósito-Escudero1,2, Chantal Durand14, Frédérique Nugues14, Cecilia Jimenez-Mallebrera1,2, Jaume Colomer1,2, Robert Y Carlier5, Hanns Lochmüller15,16,17, Susana Quijano-Roy4, Andres Nascimento1,2.   

Abstract

Mutations in the CHRNG gene cause autosomal recessive multiple pterygium syndrome (MPS). Herein we present a long-term follow-up of seven patients with CHRNG-related nonlethal MPS and we compare them with the 57 previously published patients. The objective is defining not only the clinical, histopathological, and molecular genetic characteristics, but also the type and degree of muscle involvement on whole-body magnetic resonance imaging (WBMRI). CHRNG mutations lead to a distinctive phenotype characterized by multiple congenital contractures, pterygium, and facial dysmorphism, with a stable clinical course over the years. Postnatal abnormalities at the neuromuscular junction were observed in the muscle biopsy of these patients. WBMRI showed distinctive features different from other arthrogryposis multiple congenita. A marked muscle bulk reduction is the predominant finding, mostly affecting the spinal erector muscles and gluteus maximus. Fatty infiltration was only observed in deep paravertebral muscles and distal lower limbs. Mutations in CHRNG are mainly located at the extracellular domain of the protein. Our study contributes to further define the phenotypic spectrum of CHRNG-related nonlethal MPS, including muscle imaging features, which may be useful in distinguishing it from other diffuse arthrogryposis entities.
© 2019 Wiley Periodicals, Inc.

Entities:  

Keywords:  zzm321990CHRNG; Escobar syndrome; arthrogryposis; multiple pterygium syndrome; whole-body MRI

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Year:  2019        PMID: 30868735     DOI: 10.1002/ajmg.a.61122

Source DB:  PubMed          Journal:  Am J Med Genet A        ISSN: 1552-4825            Impact factor:   2.802


  2 in total

1.  Recessive variants in COL25A1 gene as novel cause of arthrogryposis multiplex congenita with ocular congenital cranial dysinnervation disorder.

Authors:  Daniel Natera-de Benito; Julie A Jurgens; Alison Yeung; Irina T Zaharieva; Adnan Manzur; Stephanie P DiTroia; Silvio Alessandro Di Gioia; Lynn Pais; Veronica Pini; Brenda J Barry; Wai-Man Chan; James E Elder; John Christodoulou; Eleanor Hay; Eleina M England; Pinki Munot; David G Hunter; Lucy Feng; Danielle Ledoux; Anne O'Donnell-Luria; Rahul Phadke; Elizabeth C Engle; Anna Sarkozy; Francesco Muntoni
Journal:  Hum Mutat       Date:  2022-02-03       Impact factor: 4.878

2.  The Clinical and Genotypic Spectrum of Scoliosis in Multiple Pterygium Syndrome: A Case Series on 12 Children.

Authors:  Noémi Dahan-Oliel; Klaus Dieterich; Frank Rauch; Ghalib Bardai; Taylor N Blondell; Anxhela Gjyshi Gustafson; Reggie Hamdy; Xenia Latypova; Kamran Shazand; Philip F Giampietro; Harold van Bosse
Journal:  Genes (Basel)       Date:  2021-08-06       Impact factor: 4.096

  2 in total

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