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SELENON-related myopathy

MedGen UID:
1008641
Concept ID:
CN327047
Disease or Syndrome
Synonym: SEPN1-related myopathy
 
Monarch Initiative: MONDO:0100100

Definition

Myopathy caused by pathogenic variants in SELENON that is congenital or present early in childhood with neonatal hypotonia, delayed motor development, axial muscle weakness, scoliosis, and significant respiratory involvement. Spinal rigidity of varying severity is often present. [from MONDO]

Recent clinical studies

Etiology

Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, Kamsteeg EJ, Dittrich ATM, Draaisma JMT, Janssen MCH, van Engelen BGM, Erasmus CE, Voermans NC
J Neuromuscul Dis 2023;10(6):1055-1074. doi: 10.3233/JND-221673. PMID: 37807786Free PMC Article
Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, van Tilburg WCM, Buckens SCFM, Dittrich ATM, Draaisma JMT, Janssen MCH, Kamsteeg EJ, van Kleef ESB, Koene S, Smeitink JAM, Küsters B, van Tienen FHJ, Smeets HJM, van Engelen BGM, Erasmus CE, Voermans NC
BMC Neurol 2021 Aug 12;21(1):313. doi: 10.1186/s12883-021-02336-z. PMID: 34384384Free PMC Article

Diagnosis

Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, van Tilburg WCM, Buckens SCFM, Dittrich ATM, Draaisma JMT, Janssen MCH, Kamsteeg EJ, van Kleef ESB, Koene S, Smeitink JAM, Küsters B, van Tienen FHJ, Smeets HJM, van Engelen BGM, Erasmus CE, Voermans NC
BMC Neurol 2021 Aug 12;21(1):313. doi: 10.1186/s12883-021-02336-z. PMID: 34384384Free PMC Article

Therapy

Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, Kamsteeg EJ, Dittrich ATM, Draaisma JMT, Janssen MCH, van Engelen BGM, Erasmus CE, Voermans NC
J Neuromuscul Dis 2023;10(6):1055-1074. doi: 10.3233/JND-221673. PMID: 37807786Free PMC Article
Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, van Tilburg WCM, Buckens SCFM, Dittrich ATM, Draaisma JMT, Janssen MCH, Kamsteeg EJ, van Kleef ESB, Koene S, Smeitink JAM, Küsters B, van Tienen FHJ, Smeets HJM, van Engelen BGM, Erasmus CE, Voermans NC
BMC Neurol 2021 Aug 12;21(1):313. doi: 10.1186/s12883-021-02336-z. PMID: 34384384Free PMC Article

Clinical prediction guides

Bouman K, Groothuis JT, Doorduin J, van Alfen N, Udink Ten Cate FEA, van den Heuvel FMA, Nijveldt R, Kamsteeg EJ, Dittrich ATM, Draaisma JMT, Janssen MCH, van Engelen BGM, Erasmus CE, Voermans NC
J Neuromuscul Dis 2023;10(6):1055-1074. doi: 10.3233/JND-221673. PMID: 37807786Free PMC Article

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