U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Encephalopathy due to sulfite oxidase deficiency

MedGen UID:
894927
Concept ID:
C4275019
Disease or Syndrome
Synonym: encephalopathy due to sulfite oxidase deficiency
SNOMED CT: Encephalopathy due to sulfite oxidase deficiency (715980003)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Monarch Initiative: MONDO:0019358
Orphanet: ORPHA833

Definition

A rare neurometabolic disorder with features of seizures, progressive encephalopathy and lens dislocation. The prevalence is unknown but is very rare. Symptoms usually occur within the first week after birth with feeding difficulties, vomiting and seizures which are difficult to control. The majority of patients exhibit facial dysmorphism. The course is progressive, with spasticity, severe intellectual deficit, and microcephaly seen in survivors. Lens dislocation usually occurs late in infancy but has been observed as early as two months of age. A late onset form with a milder phenotype has also been described. Caused by a mutation in the SUOX gene (12q13.13). The disease follows an autosomal recessive pattern of inheritance. [from SNOMEDCT_US]

Professional guidelines

PubMed

Schwahn BC, van Spronsen F, Misko A, Pavaine J, Holmes V, Spiegel R, Schwarz G, Wong F, Horman A, Pitt J, Sass JO, Lubout C
J Inherit Metab Dis 2024 Jul;47(4):598-623. Epub 2024 Apr 16 doi: 10.1002/jimd.12730. PMID: 38627985
Cornet MC, Sands TT, Cilio MR
Semin Fetal Neonatal Med 2018 Jun;23(3):204-212. Epub 2018 Jan 31 doi: 10.1016/j.siny.2018.01.004. PMID: 29426806
Johnson JL
Prenat Diagn 2003 Jan;23(1):6-8. doi: 10.1002/pd.505. PMID: 12533804

Recent clinical studies

Etiology

Hong SY, Lin CH
Epilepsy Behav 2023 Jun;143:109246. Epub 2023 May 13 doi: 10.1016/j.yebeh.2023.109246. PMID: 37187015
Kaczmarek AT, Bahlmann N, Thaqi B, May P, Schwarz G
Mol Genet Metab 2021 Sep-Oct;134(1-2):188-194. Epub 2021 Aug 8 doi: 10.1016/j.ymgme.2021.07.011. PMID: 34420858
Kožich V, Stabler S
J Nutr 2020 Oct 1;150(Suppl 1):2506S-2517S. doi: 10.1093/jn/nxaa134. PMID: 33000152
Claerhout H, Witters P, Régal L, Jansen K, Van Hoestenberghe MR, Breckpot J, Vermeersch P
J Inherit Metab Dis 2018 Jan;41(1):101-108. Epub 2017 Oct 4 doi: 10.1007/s10545-017-0089-4. PMID: 28980090
Johnson JL
Prenat Diagn 2003 Jan;23(1):6-8. doi: 10.1002/pd.505. PMID: 12533804

Diagnosis

Schwahn BC, van Spronsen F, Misko A, Pavaine J, Holmes V, Spiegel R, Schwarz G, Wong F, Horman A, Pitt J, Sass JO, Lubout C
J Inherit Metab Dis 2024 Jul;47(4):598-623. Epub 2024 Apr 16 doi: 10.1002/jimd.12730. PMID: 38627985
Hong SY, Lin CH
Epilepsy Behav 2023 Jun;143:109246. Epub 2023 May 13 doi: 10.1016/j.yebeh.2023.109246. PMID: 37187015
Cornet MC, Sands TT, Cilio MR
Semin Fetal Neonatal Med 2018 Jun;23(3):204-212. Epub 2018 Jan 31 doi: 10.1016/j.siny.2018.01.004. PMID: 29426806
Claerhout H, Witters P, Régal L, Jansen K, Van Hoestenberghe MR, Breckpot J, Vermeersch P
J Inherit Metab Dis 2018 Jan;41(1):101-108. Epub 2017 Oct 4 doi: 10.1007/s10545-017-0089-4. PMID: 28980090
Johnson JL
Prenat Diagn 2003 Jan;23(1):6-8. doi: 10.1002/pd.505. PMID: 12533804

Therapy

Kaczmarek AT, Bahlmann N, Thaqi B, May P, Schwarz G
Mol Genet Metab 2021 Sep-Oct;134(1-2):188-194. Epub 2021 Aug 8 doi: 10.1016/j.ymgme.2021.07.011. PMID: 34420858
Wyse ATS, Grings M, Wajner M, Leipnitz G
Neurotox Res 2019 Feb;35(2):484-494. Epub 2018 Dec 5 doi: 10.1007/s12640-018-9986-z. PMID: 30515714
Cornet MC, Sands TT, Cilio MR
Semin Fetal Neonatal Med 2018 Jun;23(3):204-212. Epub 2018 Jan 31 doi: 10.1016/j.siny.2018.01.004. PMID: 29426806
Veldman A, Santamaria-Araujo JA, Sollazzo S, Pitt J, Gianello R, Yaplito-Lee J, Wong F, Ramsden CA, Reiss J, Cook I, Fairweather J, Schwarz G
Pediatrics 2010 May;125(5):e1249-54. Epub 2010 Apr 12 doi: 10.1542/peds.2009-2192. PMID: 20385644

Prognosis

Kaczmarek AT, Bahlmann N, Thaqi B, May P, Schwarz G
Mol Genet Metab 2021 Sep-Oct;134(1-2):188-194. Epub 2021 Aug 8 doi: 10.1016/j.ymgme.2021.07.011. PMID: 34420858
Claerhout H, Witters P, Régal L, Jansen K, Van Hoestenberghe MR, Breckpot J, Vermeersch P
J Inherit Metab Dis 2018 Jan;41(1):101-108. Epub 2017 Oct 4 doi: 10.1007/s10545-017-0089-4. PMID: 28980090
Westerlinck H, Meylaerts L, Van Hoestenberghe MR, Rossi A
JBR-BTR 2014 Mar-Apr;97(2):113-4. doi: 10.5334/jbr-btr.40. PMID: 25073245
Lee WT
Brain Dev 2011 Oct;33(9):745-52. Epub 2011 Jul 30 doi: 10.1016/j.braindev.2011.06.014. PMID: 21803516
Rupar CA, Gillett J, Gordon BA, Ramsay DA, Johnson JL, Garrett RM, Rajagopalan KV, Jung JH, Bacheyie GS, Sellers AR
Neuropediatrics 1996 Dec;27(6):299-304. doi: 10.1055/s-2007-973798. PMID: 9050047

Clinical prediction guides

Kaczmarek AT, Bahlmann N, Thaqi B, May P, Schwarz G
Mol Genet Metab 2021 Sep-Oct;134(1-2):188-194. Epub 2021 Aug 8 doi: 10.1016/j.ymgme.2021.07.011. PMID: 34420858
Wyse ATS, Grings M, Wajner M, Leipnitz G
Neurotox Res 2019 Feb;35(2):484-494. Epub 2018 Dec 5 doi: 10.1007/s12640-018-9986-z. PMID: 30515714
Claerhout H, Witters P, Régal L, Jansen K, Van Hoestenberghe MR, Breckpot J, Vermeersch P
J Inherit Metab Dis 2018 Jan;41(1):101-108. Epub 2017 Oct 4 doi: 10.1007/s10545-017-0089-4. PMID: 28980090
Holder JL Jr, Agadi S, Reese W, Rehder C, Quach MM
JAMA Neurol 2014 Jun;71(6):782-4. doi: 10.1001/jamaneurol.2013.5083. PMID: 24756183
Tan WH, Eichler FS, Hoda S, Lee MS, Baris H, Hanley CA, Grant PE, Krishnamoorthy KS, Shih VE
Pediatrics 2005 Sep;116(3):757-66. doi: 10.1542/peds.2004-1897. PMID: 16140720

Recent systematic reviews

Schwahn BC, van Spronsen F, Misko A, Pavaine J, Holmes V, Spiegel R, Schwarz G, Wong F, Horman A, Pitt J, Sass JO, Lubout C
J Inherit Metab Dis 2024 Jul;47(4):598-623. Epub 2024 Apr 16 doi: 10.1002/jimd.12730. PMID: 38627985

Supplemental Content

Table of contents

    Clinical resources

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Recent activity

    Your browsing activity is empty.

    Activity recording is turned off.

    Turn recording back on

    See more...