Coexistence of junctional epidermolysis bullosa, autosomal recessive deafness type 57, and Angelman syndrome: A case report.

Angelman syndrome COL17A1 PDZD7 UBE3A autosomal recessive deafness type 57 case report epidermolysis bullosa

Journal

Clinical case reports
ISSN: 2050-0904
Titre abrégé: Clin Case Rep
Pays: England
ID NLM: 101620385

Informations de publication

Date de publication:
Apr 2023
Historique:
received: 14 09 2022
revised: 18 03 2023
accepted: 12 04 2023
medline: 28 4 2023
pubmed: 28 4 2023
entrez: 28 4 2023
Statut: epublish

Résumé

The presence of more than one genetic/genomic disorder is not uncommon. It is therefore essential to continuously consider new signs and symptoms over time. Administration of gene therapy could be extremely difficult in particular situations. A 9-month-old boy presented to our department for evaluation of developmental delay. We found that he was affected by intermediate junctional epidermolysis bullosa (COL17A1, c.3766 + 1G > A, homozygous), Angelman syndrome (5,5 Mb deletion of 15q11.2-q13.1), and autosomal recessive deafness type 57 (PDZD7, c.883C > T, homozygous). This case report highlights the value of reanalyzing genetic data in patients with rare diseases and developmental delay. It also emphasizes the importance of considering potential superimposed diagnoses in individuals with multiple genetic variants. Early diagnosis and intervention can improve outcomes and prevent further complications in affected patients.

Autres résumés

Type: Publisher (rus)
This case report highlights the value of reanalyzing genetic data in patients with rare diseases and developmental delay. It also emphasizes the importance of considering potential superimposed diagnoses in individuals with multiple genetic variants. Early diagnosis and intervention can improve outcomes and prevent further complications in affected patients.

Identifiants

pubmed: 37113642
doi: 10.1002/ccr3.7275
pii: CCR37275
pmc: PMC10126752
doi:

Types de publication

Case Reports

Langues

eng

Pagination

e7275

Informations de copyright

© 2023 The Authors. Clinical Case Reports published by John Wiley & Sons Ltd.

Déclaration de conflit d'intérêts

The authors have no conflict of interest to declare.

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Auteurs

Maria Eugenia Amato (ME)

Pediatric Neurology Department, Institut de Recerca Hospital Sant Joan de Déu Barcelona Barcelona Spain.

Silvia Ricart (S)

Chronic Complex and Palliative Pediatric Service Hospital Sant Joan de Déu Barcelona Barcelona Spain.

Maria Asunción Vicente (MA)

Department of Dermatology Hospital Sant Joan de Déu Barcelona Spain.
European Reference Network for rare skin diseases (ERN-Skin) Barcelona Spain.
CSUR Epidermolisis Ampollosa Hereditaria Barcelona Spain.

Loreto Martorell (L)

Department of Genetic and Molecular Medicine-IPER Institut de Recerca Sant Joan de Déu Barcelona Spain.
U-703 Centre for Biomedical Research on Rare Diseases (CIBER-ER) Instituto de Salud Carlos III Barcelona Spain.

Judith Armstrong (J)

Department of Genetic and Molecular Medicine-IPER Institut de Recerca Sant Joan de Déu Barcelona Spain.
U-703 Centre for Biomedical Research on Rare Diseases (CIBER-ER) Instituto de Salud Carlos III Barcelona Spain.

Guerau Fernández Isern (G)

Department of Genetic and Molecular Medicine-IPER Institut de Recerca Sant Joan de Déu Barcelona Spain.

José Manuel Mascaro (JM)

Department of Dermatology Hospital Clinic Barcelona Spain.

Sol Balsells (S)

Department of Statistics Institut de Recerca Sant Joan de Déu Barcelona Spain.

Itziar Alonso (I)

Pediatric Neurology Department, Institut de Recerca Hospital Sant Joan de Déu Barcelona Barcelona Spain.

Mercedes Serrano (M)

Pediatric Neurology Department, Institut de Recerca Hospital Sant Joan de Déu Barcelona Barcelona Spain.
U-703 Centre for Biomedical Research on Rare Diseases (CIBER-ER) Instituto de Salud Carlos III Barcelona Spain.

Juan Darío Ortigoza-Escobar (JD)

U-703 Centre for Biomedical Research on Rare Diseases (CIBER-ER) Instituto de Salud Carlos III Barcelona Spain.
Movement Disorders Unit, Pediatric Neurology Department Institut de Recerca, Hospital Sant Joan de Déu Barcelona Barcelona Spain.
European Reference Network for Rare Neurological Diseases (ERN-RND) Barcelona Spain.

Classifications MeSH