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@ARTICLE{Wunderlich:857100,
author = {Wunderlich, G. and Brunn, A. and Daimagüler, H.-S. and
Bozoglu, T. and Fink, G. R. and Lehmann, H. C. and Weis, J.
and Cirak, S.},
title = {{L}ong term history of a congenital core-rod myopathy with
compound heterozygous mutations in the {N}ebulin gene},
journal = {Acta myologica},
volume = {37},
number = {2},
issn = {1128-2460},
address = {Ospedaletto (PI)},
publisher = {Pacini},
reportid = {FZJ-2018-06354},
pages = {121 - 127},
year = {2018},
abstract = {Mutations in the Nebulin gene (NEB) may cause core-rod
myopathy. The large size of the gene so far prevented
inclusion of its routine analysis by didesoxy resequencing
methodology in the diagnostic regime for muscular dystrophy
cases. Here we report a 54-year-old female with a rare
histological myopathy presentation of co-occurring cores and
rods. The patient reported early childhood onset weakness.
Muscle-MRI showed mainly proximal muscle involvement. We
identified two compound heterozygous non-sense mutations in
NEB (c.19653G > A, p.W6551* exon 127 and c.25441C > T,
p.R8481* exon 182) using a comprehensive next generation
sequencing (NGS)-based approach named Mendeliome Sequencing.
The p.W6551* mutation has not been reported elsewhere. Early
diagnosis by NGS shall be chased since even a scoliosis
surgery at the age of 18 years had failed to initiate a
neurological workup. Rather, cosmetic surgery for facial
weakness had been performed recently, albeit with an
unsatisfactory outcome},
cin = {INM-3},
ddc = {610},
cid = {I:(DE-Juel1)INM-3-20090406},
pnm = {572 - (Dys-)function and Plasticity (POF3-572)},
pid = {G:(DE-HGF)POF3-572},
typ = {PUB:(DE-HGF)16},
url = {https://juser.fz-juelich.de/record/857100},
}