Literature DB >> 31060723

Signs and Symptoms in Congenital Myopathies.

Hernan D Gonorazky1, James J Dowling2, Jonathan R Volpatti3, Jiri Vajsar4.   

Abstract

Congenital myopathies (CM) represent a continuously growing group of disorders with a wide range of clinical and histopathologic presentations. The refinement and application of new technologies for genetic diagnosis have broadened our understanding of the genetic causes of CM. Our growing knowledge has revealed that there are no clear limits between each subgroup of CM, and thus the clinical overlap between genes has become more evident. The implementation of next generation sequencing has produced vast amounts of genomic data that may be difficult to interpret. With an increasing number of reports revealing variants of unknown significance, it is essential to support the genetic diagnosis with a well characterized clinical description of the patient. Phenotype-genotype correlation should be a priority at the moment of disclosing the genetic results. Thus, a detailed physical examination can provide us with subtle differences that are not only key in order to arrive at a correct diagnosis, but also in the characterization of new myopathies and candidate genes.
Copyright © 2019 Elsevier Inc. All rights reserved.

Entities:  

Mesh:

Year:  2019        PMID: 31060723     DOI: 10.1016/j.spen.2019.01.002

Source DB:  PubMed          Journal:  Semin Pediatr Neurol        ISSN: 1071-9091            Impact factor:   1.636


  2 in total

1.  Biallelic LINE insertion mutation in HACD1 causing congenital myopathy.

Authors:  Fatema Al Amrani; Carolina Gorodetsky; Lili-Naz Hazrati; Kimberly Amburgey; Hernan D Gonorazky; James J Dowling
Journal:  Neurol Genet       Date:  2020-04-13

2.  Targeted transcript analysis in muscles from patients with genetically diverse congenital myopathies.

Authors:  Christoph Bachmann; Martina Franchini; Luuk R Van den Bersselaar; Nick Kruijt; Nicol C Voermans; Karlijn Bouman; Erik-Jan Kamsteeg; Karl Christian Knop; Lucia Ruggiero; Lucio Santoro; Yoram Nevo; Jo Wilmshurst; John Vissing; Michael Sinnreich; Daniele Zorzato; Francesco Muntoni; Heinz Jungbluth; Francesco Zorzato; Susan Treves
Journal:  Brain Commun       Date:  2022-09-02
  2 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.