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Severe phosphoribosylpyrophosphate synthetase superactivity

MedGen UID:
1843045
Concept ID:
C5680017
Disease or Syndrome
Synonyms: severe phosphoribosylpyrophosphate synthetase superactivity; Severe PRPP synthetase superactivity; severe PRPP synthetase superactivity; Severe PRPS1 superactivity; severe PRPS1 superactivity
Modes of inheritance:
X-linked recessive inheritance
MedGen UID:
375779
Concept ID:
C1845977
Finding
Source: Orphanet
A mode of inheritance that is observed for recessive traits related to a gene encoded on the X chromosome. In the context of medical genetics, X-linked recessive disorders manifest in males (who have one copy of the X chromosome and are thus hemizygotes), but generally not in female heterozygotes who have one mutant and one normal allele.
 
Monarch Initiative: MONDO:0018464
Orphanet: ORPHA411543

Definition

A severe form of phosphoribosylpyrophosphate (PRPP) synthetase superactivity, an X-linked disorder of purine metabolism, characterized by early onset hyperuricemia and hyperuricosuria, and clinically manifesting with urolithiasis, gout and neurodevelopmental anomalies consisting of variable combinations of sensorineural hearing loss, hypotonia, and ataxia. [from ORDO]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVSevere phosphoribosylpyrophosphate synthetase superactivity

Recent clinical studies

Diagnosis

Zikánová M, Wahezi D, Hay A, Stiburková B, Pitts C 3rd, Mušálková D, Škopová V, Barešová V, Soucková O, Hodanová K, Živná M, Stránecký V, Hartmannová H, Hnízda A, Bleyer AJ, Kmoch S
Rheumatology (Oxford) 2018 Jul 1;57(7):1180-1185. doi: 10.1093/rheumatology/key041. PMID: 30423175Free PMC Article

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