Literature DB >> 11571700

Reducing body myopathy with cytoplasmic bodies and rigid spine syndrome: a mixed congenital myopathy.

H H Goebel1, L E Halbig, L Goldfarb, R Schober, M Albani, E Neuen-Jacob, T Voit.   

Abstract

At the age of five years a male child started to develop a progressive rigid spine, torsion scoliosis, and flexion contractures of his elbows, knees, hips, and ankles owing to severe proximal and distal muscle weakness. He had three muscle biopsies from three different muscles at ages 7, 11, and 14 years, respectively. Myopathologically, these muscle tissues contained numerous inclusions which, at the ultrastructural level, turned out to be reducing bodies and cytoplasmic bodies, often in close spatial proximity. Similar histological inclusions, although not further identified by histochemistry and electron microscopy, were seen in his maternal grandmother's biopsied muscle tissue who had developed weakness of the legs and hands after the age of 50 years. The patient's parents were healthy, but the mother's quadriceps muscle showed an increased spectrum of muscle fibre diameters. Our patient, thus, had a neuromuscular disorder, perhaps familial, presenting as a mixed congenital myopathy, i.e., reducing body myopathy with cytoplasmic bodies, of which the morphological lesions could be consistently documented over several years in his different limb muscles. While other mixed congenital myopathies had shown cores and rods, both related to sarcomeres and thus possibly morphogenetically related, cytoplasmic bodies thought to be related to Z-bands and reducing bodies dissimilar to any muscle fibre constituent do not share any common denominator. Therefore, we suggest that this neuromuscular disorder may be a unique mixed congenital myopathy, either sporadic or genetic. In the latter case, the transmission pattern suggested X-linked recessive inheritance, but an autosomal-dominant transmission with variable penetrance could not be ruled out.

Entities:  

Mesh:

Year:  2001        PMID: 11571700     DOI: 10.1055/s-2001-17374

Source DB:  PubMed          Journal:  Neuropediatrics        ISSN: 0174-304X            Impact factor:   1.947


  5 in total

Review 1.  Myofibrillar myopathies: new developments.

Authors:  Montse Olivé; Rudolf A Kley; Lev G Goldfarb
Journal:  Curr Opin Neurol       Date:  2013-10       Impact factor: 5.710

2.  169th ENMC International Workshop Rare Structural Congenital Myopathies 6-8 November 2009, Naarden, The Netherlands.

Authors:  Hans H Goebel; Carsten G Bönnemann
Journal:  Neuromuscul Disord       Date:  2011-03-29       Impact factor: 4.296

3.  199th ENMC international workshop: FHL1 related myopathies, June 7-9, 2013, Naarden, The Netherlands.

Authors:  Anne T Bertrand; Carsten G Bönnemann; Gisèle Bonne
Journal:  Neuromuscul Disord       Date:  2014-02-14       Impact factor: 4.296

4.  Clinical, histological and genetic characterization of reducing body myopathy caused by mutations in FHL1.

Authors:  Joachim Schessl; Ana L Taratuto; Caroline Sewry; Roberta Battini; Steven S Chin; Baijayanta Maiti; Alberto L Dubrovsky; Marcela G Erro; Graciela Espada; Monica Robertella; Maria Saccoliti; Patricia Olmos; Leslie R Bridges; Peter Standring; Ying Hu; Yaqun Zou; Kathryn J Swoboda; Mena Scavina; Hans-Hilmar Goebel; Christina A Mitchell; Kevin M Flanigan; Francesco Muntoni; Carsten G Bönnemann
Journal:  Brain       Date:  2009-01-29       Impact factor: 13.501

5.  Proteomic identification of FHL1 as the protein mutated in human reducing body myopathy.

Authors:  Joachim Schessl; Yaqun Zou; Meagan J McGrath; Belinda S Cowling; Baijayanta Maiti; Steven S Chin; Caroline Sewry; Roberta Battini; Ying Hu; Denny L Cottle; Michael Rosenblatt; Lynn Spruce; Arupa Ganguly; Janbernd Kirschner; Alexander R Judkins; Jeffrey A Golden; Hans-Hilmar Goebel; Francesco Muntoni; Kevin M Flanigan; Christina A Mitchell; Carsten G Bönnemann
Journal:  J Clin Invest       Date:  2008-03       Impact factor: 14.808

  5 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.