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Alport syndrome with mental retardation midface hypoplasia & elliptocytosis

Pathology: - defect in type 4 collagen [3] - disease affects basement membranes Genetics: - X-linked contiguous gene deletion syndrome - associated with defects in AMMECR1 - associated with defects in ACSL4 Clinical manifestations: - glomerulonephritis - sensorineural deafness - mental retardation - midface hypoplasia - ocular abnormalities Laboratory: - peripheral blood smear: elliptocytosis - renal function testing - urinalysis - proteinuria - hematuria Management: - renal transplantation for end-stage renal disease - underlying disease for not recur in renal transplant [2]

General

Alport syndrome; hereditary nephropathy genetic syndrome (multisystem disorder)

Database Correlations

OMIM 300194

References

  1. OMIM :accession 300194
  2. Medical Knowledge Self Assessment Program (MKSAP) 16, 18. American College of Physicians, Philadelphia 2012, 2018.
  3. Gubler MC. Inherited diseases of the glomerular basement membrane. Nat Clin Pract Nephrol. 2008 Jan;4(1):24-37. PMID: 18094725