Clinical, Genetic, and Histological Characterization of Patients with Rare Neuromuscular and Mitochondrial Diseases Presenting with Different Cardiomyopathy Phenotypes.


Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
22 May 2023
Historique:
received: 20 04 2023
revised: 15 05 2023
accepted: 20 05 2023
medline: 29 5 2023
pubmed: 27 5 2023
entrez: 27 5 2023
Statut: epublish

Résumé

Cardiomyopathies are mostly determined by genetic mutations affecting either cardiac muscle cell structure or function. Nevertheless, cardiomyopathies may also be part of complex clinical phenotypes in the spectrum of neuromuscular (NMD) or mitochondrial diseases (MD). The aim of this study is to describe the clinical, molecular, and histological characteristics of a consecutive cohort of patients with cardiomyopathy associated with NMDs or MDs referred to a tertiary cardiomyopathy clinic. Consecutive patients with a definitive diagnosis of NMDs and MDs presenting with a cardiomyopathy phenotype were described. Seven patients were identified: two patients with ACAD9 deficiency (

Identifiants

pubmed: 37240454
pii: ijms24109108
doi: 10.3390/ijms24109108
pmc: PMC10218839
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

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Auteurs

Emanuele Monda (E)

Inherited and Rare Cardiovascular Disease Unit, Department of Translational Medical Sciences, University of Campania Luigi Vanvitelli, AORN dei Colli, Monaldi Hospital, 81031 Naples, Italy.

Michele Lioncino (M)

Inherited and Rare Cardiovascular Disease Unit, Department of Translational Medical Sciences, University of Campania Luigi Vanvitelli, AORN dei Colli, Monaldi Hospital, 81031 Naples, Italy.

Martina Caiazza (M)

Inherited and Rare Cardiovascular Disease Unit, Department of Translational Medical Sciences, University of Campania Luigi Vanvitelli, AORN dei Colli, Monaldi Hospital, 81031 Naples, Italy.

Vincenzo Simonelli (V)

Neurology Unit, AORN dei Colli, Monaldi Hospital, 81031 Naples, Italy.

Claudia Nesti (C)

Molecular Medicine, IRCCS Stella Maris Foundation, 56128 Pisa, Italy.

Marta Rubino (M)

Inherited and Rare Cardiovascular Disease Unit, Department of Translational Medical Sciences, University of Campania Luigi Vanvitelli, AORN dei Colli, Monaldi Hospital, 81031 Naples, Italy.

Alessia Perna (A)

Inherited and Rare Cardiovascular Disease Unit, Department of Translational Medical Sciences, University of Campania Luigi Vanvitelli, AORN dei Colli, Monaldi Hospital, 81031 Naples, Italy.

Alfredo Mauriello (A)

Inherited and Rare Cardiovascular Disease Unit, Department of Translational Medical Sciences, University of Campania Luigi Vanvitelli, AORN dei Colli, Monaldi Hospital, 81031 Naples, Italy.

Alberta Budillon (A)

Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, Center for Rare Diseases and InterUniversity Center for Research in Neurosciences, University of Campania Luigi Vanvitelli, Via Sergio Pansini, 5, 80131 Naples, Italy.

Vincenzo Pota (V)

NeuroMuscular Omnicentre (NEMO), AORN dei Colli, Monaldi Hospital, 80131 Naples, Italy.

Giorgia Bruno (G)

Pediatric Neurology Unit, Department of Neurosciences, Santobono-Pausilipon Children's Hospital, 80122 Naples, Italy.

Antonio Varone (A)

Pediatric Neurology Unit, Department of Neurosciences, Santobono-Pausilipon Children's Hospital, 80122 Naples, Italy.

Vincenzo Nigro (V)

Department of Precision Medicine, University of Campania Luigi Vanvitelli, Via Luigi De Crecchio 7, 80138 Naples, Italy.
Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, 80078 Pozzuoli, Italy.

Filippo Maria Santorelli (FM)

Molecular Medicine, IRCCS Stella Maris Foundation, 56128 Pisa, Italy.

Giuseppe Pacileo (G)

Heart Failure Unit, Department of Cardiology, AORN dei Colli, Monaldi Hospital, 80131 Naples, Italy.

Maria Giovanna Russo (MG)

Paediatric Cardiology Unit, University of Campania Luigi Vanvitelli, AORN dei Colli, Monaldi Hospital, 81100 Caserta, Italy.

Giulia Frisso (G)

Department of Molecular Medicine and Medical Biotechnology, University of Naples "Federico II", 80138 Naples, Italy.

Simone Sampaolo (S)

Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, Center for Rare Diseases and InterUniversity Center for Research in Neurosciences, University of Campania Luigi Vanvitelli, Via Sergio Pansini, 5, 80131 Naples, Italy.

Giuseppe Limongelli (G)

Inherited and Rare Cardiovascular Disease Unit, Department of Translational Medical Sciences, University of Campania Luigi Vanvitelli, AORN dei Colli, Monaldi Hospital, 81031 Naples, Italy.
NeuroMuscular Omnicentre (NEMO), AORN dei Colli, Monaldi Hospital, 80131 Naples, Italy.
Institute of Cardiovascular Sciences, University College of London and St. Bartholomew's Hospital, Gower St, London WC1E 6DD, UK.

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