A novel

Cardiomyopathy Lipid metabolism Neutral lipid storage disease with myopathy PNPLA2 Splicing mutation Triglyceride lipase

Journal

Genes & diseases
ISSN: 2352-3042
Titre abrégé: Genes Dis
Pays: Netherlands
ID NLM: 101635967

Informations de publication

Date de publication:
Jan 2021
Historique:
received: 20 06 2019
revised: 12 07 2019
accepted: 22 07 2019
entrez: 11 2 2021
pubmed: 29 7 2019
medline: 29 7 2019
Statut: epublish

Résumé

Neutral lipid storage disease with myopathy (NLSDM) is a rare autosomal recessive disorder, due to an enzymatic error of lipid metabolism. Patients present always with skeletal muscle myopathy and variable cardiac and hepatic involvement. NLSDM is caused by mutations in the

Identifiants

pubmed: 33569515
doi: 10.1016/j.gendis.2019.07.006
pii: S2352-3042(19)30050-9
pmc: PMC7859421
doi:

Types de publication

Journal Article

Langues

eng

Pagination

73-78

Informations de copyright

© 2019 Chongqing Medical University. Production and hosting by Elsevier B.V.

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Auteurs

Daniela Tavian (D)

Laboratory of Cellular Biochemistry and Molecular Biology, CRIBENS, Università Cattolica del Sacro Cuore, pz Buonarroti 30, Milan, 20145, Italy.
Department of Psychology, Università Cattolica del Sacro Cuore, Largo Gemelli 1, Milan, 20123, Italy.

Lorenzo Maggi (L)

Neuromuscular Diseases and Neuroimmunology Unit, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Marina Mora (M)

Neuromuscular Diseases and Neuroimmunology Unit, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Lucia Morandi (L)

Neuromuscular Diseases and Neuroimmunology Unit, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.

Cinzia Bragato (C)

Neuromuscular Diseases and Neuroimmunology Unit, Fondazione IRCCS Istituto Neurologico "Carlo Besta", Milan, Italy.
PhD program in Neuroscience, University of Milano-Bicocca, Via Cadore 48, Monza, 20900, Italy.

Sara Missaglia (S)

Laboratory of Cellular Biochemistry and Molecular Biology, CRIBENS, Università Cattolica del Sacro Cuore, pz Buonarroti 30, Milan, 20145, Italy.
Department of Psychology, Università Cattolica del Sacro Cuore, Largo Gemelli 1, Milan, 20123, Italy.

Classifications MeSH