Literature DB >> 31227654

Dihydropyridine Receptor Congenital Myopathy In A Consangineous Turkish Family.

Uluç Yiş1, Semra Hiz1, Sezgin Güneş2, Gülden Diniz3, Figen Baydan3, Ana Töpf4, Ece Sonmezler5, Hanns Lochmüller6, Rita Horvath7, Yavuz Oktay5,8,9.   

Abstract

Dihydropyridine receptor congenital myopathy is a recently described congenital myopathy caused by dominant or recessive mutations in the CACNA1S gene. To date, only 11 cases from 7 families were described in a single report. Here, we describe a consanguineous family with three affected children, presenting congenital hypotonia, contractures, ophthalmoplegia and respiratory insufficiency, with a novel homozygous mutation in the CACNA1S gene. They also showed cognitive delay, pes equinovarus deformity and neurogenic changes that have not been associated with this myopathy in the previous reports. This report expands the phenotypic spectrum of dihydropyridine receptor congenital myopathy and underscores the importance of whole exome sequencing in early onset neuromuscular disorders.

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Year:  2019        PMID: 31227654      PMCID: PMC6736676          DOI: 10.3233/JND-190383

Source DB:  PubMed          Journal:  J Neuromuscul Dis


  15 in total

Review 1.  Skeletal muscle excitation-contraction coupling: who are the dancing partners?

Authors:  Robyn T Rebbeck; Yamuna Karunasekara; Philip G Board; Nicole A Beard; Marco G Casarotto; Angela F Dulhunty
Journal:  Int J Biochem Cell Biol       Date:  2013-12-24       Impact factor: 5.085

Review 2.  Ion channel voltage sensors: structure, function, and pathophysiology.

Authors:  William A Catterall
Journal:  Neuron       Date:  2010-09-23       Impact factor: 17.173

3.  Dihydropyridine receptor (DHPR, CACNA1S) congenital myopathy.

Authors:  Vanessa Schartner; Norma B Romero; Sandra Donkervoort; Susan Treves; Pinki Munot; Tyler Mark Pierson; Ivana Dabaj; Edoardo Malfatti; Irina T Zaharieva; Francesco Zorzato; Osorio Abath Neto; Guy Brochier; Xavière Lornage; Bruno Eymard; Ana Lía Taratuto; Johann Böhm; Hernan Gonorazky; Leigh Ramos-Platt; Lucy Feng; Rahul Phadke; Diana X Bharucha-Goebel; Charlotte Jane Sumner; Mai Thao Bui; Emmanuelle Lacene; Maud Beuvin; Clémence Labasse; Nicolas Dondaine; Raphael Schneider; Julie Thompson; Anne Boland; Jean-François Deleuze; Emma Matthews; Aleksandra Nadaj Pakleza; Caroline A Sewry; Valérie Biancalana; Susana Quijano-Roy; Francesco Muntoni; Michel Fardeau; Carsten G Bönnemann; Jocelyn Laporte
Journal:  Acta Neuropathol       Date:  2016-12-23       Impact factor: 17.088

Review 4.  Pharmacogenetics and pathophysiology of CACNA1S mutations in malignant hyperthermia.

Authors:  Teresa A Beam; Emily F Loudermilk; David F Kisor
Journal:  Physiol Genomics       Date:  2016-12-23       Impact factor: 3.107

5.  Stac adaptor proteins regulate trafficking and function of muscle and neuronal L-type Ca2+ channels.

Authors:  Alexander Polster; Stefano Perni; Hicham Bichraoui; Kurt G Beam
Journal:  Proc Natl Acad Sci U S A       Date:  2014-12-29       Impact factor: 11.205

6.  Association of novel single nucleotide polymorphisms in the calcium channel alpha 1 subunit gene (Ca(v)1.1) and thyrotoxic periodic paralysis.

Authors:  Annie W C Kung; K S Lau; G C Y Fong; Vivian Chan
Journal:  J Clin Endocrinol Metab       Date:  2004-03       Impact factor: 5.958

Review 7.  Ca(V)1.1: The atypical prototypical voltage-gated Ca²⁺ channel.

Authors:  Roger A Bannister; Kurt G Beam
Journal:  Biochim Biophys Acta       Date:  2012-09-13

8.  Approach to the diagnosis of congenital myopathies.

Authors:  Kathryn N North; Ching H Wang; Nigel Clarke; Heinz Jungbluth; Mariz Vainzof; James J Dowling; Kimberly Amburgey; Susana Quijano-Roy; Alan H Beggs; Caroline Sewry; Nigel G Laing; Carsten G Bönnemann
Journal:  Neuromuscul Disord       Date:  2013-11-18       Impact factor: 4.296

9.  STAC3 stably interacts through its C1 domain with CaV1.1 in skeletal muscle triads.

Authors:  Marta Campiglio; Bernhard E Flucher
Journal:  Sci Rep       Date:  2017-01-23       Impact factor: 4.379

10.  Voltage sensor charge loss accounts for most cases of hypokalemic periodic paralysis.

Authors:  E Matthews; R Labrum; M G Sweeney; R Sud; A Haworth; P F Chinnery; G Meola; S Schorge; D M Kullmann; M B Davis; M G Hanna
Journal:  Neurology       Date:  2008-12-31       Impact factor: 9.910

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  3 in total

1.  Targeted Therapies for Skeletal Muscle Ion Channelopathies: Systematic Review and Steps Towards Precision Medicine.

Authors:  Jean-François Desaphy; Concetta Altamura; Savine Vicart; Bertrand Fontaine
Journal:  J Neuromuscul Dis       Date:  2021

Review 2.  Update on Congenital Myopathies in Adulthood.

Authors:  George Konstantinos Papadimas; Sophia Xirou; Evangelia Kararizou; Constantinos Papadopoulos
Journal:  Int J Mol Sci       Date:  2020-05-24       Impact factor: 5.923

Review 3.  Mutations in proteins involved in E-C coupling and SOCE and congenital myopathies.

Authors:  Daniela Rossi; Maria Rosaria Catallo; Enrico Pierantozzi; Vincenzo Sorrentino
Journal:  J Gen Physiol       Date:  2022-08-18       Impact factor: 4.000

  3 in total

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