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Galloway-Mowat syndrome 8(GAMOS8)

MedGen UID:
1675829
Concept ID:
C5193045
Disease or Syndrome
Synonyms: GALLOWAY-MOWAT SYNDROME 8; GAMOS8
 
Gene (location): NUP133 (1q42.13)
 
Monarch Initiative: MONDO:0032693
OMIM®: 618349

Definition

Galloway-Mowat syndrome-8 (GAMOS8) is an autosomal recessive disorder characterized by impaired psychomotor development, poor overall growth with microcephaly, and early-onset progressive nephrotic syndrome associated with focal segmental glomerulosclerosis on renal biopsy. Some patients may have seizures, and some may die in childhood (summary by Fujita et al., 2018). For a general phenotypic description and a discussion of genetic heterogeneity of GAMOS, see GAMOS1 (251300). [from OMIM]

Clinical features

From HPO
Focal segmental glomerulosclerosis
MedGen UID:
4904
Concept ID:
C0017668
Disease or Syndrome
Segmental accumulation of scar tissue in individual (but not all) glomeruli.
Hematuria
MedGen UID:
5488
Concept ID:
C0018965
Disease or Syndrome
The presence of blood in the urine. Hematuria may be gross hematuria (visible to the naked eye) or microscopic hematuria (detected by dipstick or microscopic examination of the urine).
Nephrotic syndrome
MedGen UID:
10308
Concept ID:
C0027726
Disease or Syndrome
Nephrotic syndrome is a collection of findings resulting from glomerular dysfunction with an increase in glomerular capillary wall permeability associated with pronounced proteinuria. Nephrotic syndrome refers to the constellation of clinical findings that result from severe renal loss of protein, with Proteinuria and hypoalbuminemia, edema, and hyperlipidemia.
Proteinuria
MedGen UID:
10976
Concept ID:
C0033687
Finding
Increased levels of protein in the urine.
Renal tubular atrophy
MedGen UID:
388054
Concept ID:
C1858395
Finding
The presence of renal tubules with thick redundant basement membranes, or a reduction of greater than 50% in tubular diameter compared to surrounding non-atrophic tubules.
Stage 5 chronic kidney disease
MedGen UID:
384526
Concept ID:
C2316810
Disease or Syndrome
A degree of kidney failure severe enough to require dialysis or kidney transplantation for survival characterized by a severe reduction in glomerular filtration rate (less than 15 ml/min/1.73 m2) and other manifestations including increased serum creatinine.
Hearing impairment
MedGen UID:
235586
Concept ID:
C1384666
Disease or Syndrome
A decreased magnitude of the sensory perception of sound.
Seizure
MedGen UID:
20693
Concept ID:
C0036572
Sign or Symptom
A seizure is an intermittent abnormality of nervous system physiology characterized by a transient occurrence of signs and/or symptoms due to abnormal excessive or synchronous neuronal activity in the brain.
Global developmental delay
MedGen UID:
107838
Concept ID:
C0557874
Finding
A delay in the achievement of motor or mental milestones in the domains of development of a child, including motor skills, speech and language, cognitive skills, and social and emotional skills. This term should only be used to describe children younger than five years of age.
Abnormal cerebellum morphology
MedGen UID:
400925
Concept ID:
C1866129
Anatomical Abnormality
Any structural abnormality of the cerebellum.
Focal cortical dysplasia
MedGen UID:
853938
Concept ID:
C2938983
Congenital Abnormality
A type of malformation of cortical development that primarily affects areas of neocortex. It can be identified on conventional magnetic resonance imaging as focal cortical thickening, abnormal gyration, and blurring between gray and white matter, often associated with clusters of heterotopic neurons.
Intellectual disability, profound
MedGen UID:
892508
Concept ID:
C3161330
Mental or Behavioral Dysfunction
Profound mental retardation is defined as an intelligence quotient (IQ) below 20.
Delayed CNS myelination
MedGen UID:
867393
Concept ID:
C4021758
Anatomical Abnormality
Delayed myelination in the central nervous system.
Cerebral cortical atrophy
MedGen UID:
1646740
Concept ID:
C4551583
Disease or Syndrome
Atrophy of the cortex of the cerebrum.
Brain atrophy
MedGen UID:
1643639
Concept ID:
C4551584
Disease or Syndrome
Partial or complete wasting (loss) of brain tissue that was once present.
Hypotonia
MedGen UID:
10133
Concept ID:
C0026827
Finding
Hypotonia is an abnormally low muscle tone (the amount of tension or resistance to movement in a muscle). Even when relaxed, muscles have a continuous and passive partial contraction which provides some resistance to passive stretching. Hypotonia thus manifests as diminished resistance to passive stretching. Hypotonia is not the same as muscle weakness, although the two conditions can co-exist.
Microcephaly
MedGen UID:
1644158
Concept ID:
C4551563
Finding
Head circumference below 2 standard deviations below the mean for age and gender.
Hypoalbuminemia
MedGen UID:
68694
Concept ID:
C0239981
Finding
Reduction in the concentration of albumin in the blood.
Enamel hypoplasia
MedGen UID:
3730
Concept ID:
C0011351
Disease or Syndrome
Developmental hypoplasia of the dental enamel.
Narrow forehead
MedGen UID:
326956
Concept ID:
C1839758
Finding
Width of the forehead or distance between the frontotemporales is more than two standard deviations below the mean (objective); or apparently narrow intertemporal region (subjective).
Strabismus
MedGen UID:
21337
Concept ID:
C0038379
Disease or Syndrome
A misalignment of the eyes so that the visual axes deviate from bifoveal fixation. The classification of strabismus may be based on a number of features including the relative position of the eyes, whether the deviation is latent or manifest, intermittent or constant, concomitant or otherwise and according to the age of onset and the relevance of any associated refractive error.

Recent clinical studies

Etiology

Sinha R, Vasudevan A, Agarwal I, Sethi SK, Saha A, Pradhan S, Ekambaram S, Thaker N, Matnani M, Banerjee S, Sharma J, Singhal J, Ashraf S, Mandal K
Nephron 2020;144(1):21-29. Epub 2019 Oct 25 doi: 10.1159/000503303. PMID: 31655822

Diagnosis

Alves GR, Javaroni JB, Moura APGE, Consolaro A, Segato RAB
Spec Care Dentist 2024 Jul-Aug;44(4):1054-1058. Epub 2024 Feb 6 doi: 10.1111/scd.12971. PMID: 38321585
Racine J, Golden R
Doc Ophthalmol 2021 Aug;143(1):75-83. Epub 2021 Feb 6 doi: 10.1007/s10633-021-09820-4. PMID: 33548032
Sinha R, Vasudevan A, Agarwal I, Sethi SK, Saha A, Pradhan S, Ekambaram S, Thaker N, Matnani M, Banerjee S, Sharma J, Singhal J, Ashraf S, Mandal K
Nephron 2020;144(1):21-29. Epub 2019 Oct 25 doi: 10.1159/000503303. PMID: 31655822
Krishnamurthy S, Rajesh NG, Ramesh A, Zenker M
Indian J Pediatr 2012 Aug;79(8):1087-90. Epub 2011 Dec 3 doi: 10.1007/s12098-011-0616-5. PMID: 22144119
Lin CC, Tsai JD, Lin SP, Tzen CY, Shen EY, Shih CS
Pediatr Nephrol 2001 Aug;16(8):653-7. doi: 10.1007/s004670100628. PMID: 11519896

Prognosis

Krishnamurthy S, Rajesh NG, Ramesh A, Zenker M
Indian J Pediatr 2012 Aug;79(8):1087-90. Epub 2011 Dec 3 doi: 10.1007/s12098-011-0616-5. PMID: 22144119
Shiihara T, Kato M, Kimura T, Matsunaga A, Joh K, Hayasaka K
J Child Neurol 2003 Feb;18(2):147-9. doi: 10.1177/08830738030180021801. PMID: 12693786

Clinical prediction guides

Alves GR, Javaroni JB, Moura APGE, Consolaro A, Segato RAB
Spec Care Dentist 2024 Jul-Aug;44(4):1054-1058. Epub 2024 Feb 6 doi: 10.1111/scd.12971. PMID: 38321585
Lin CC, Tsai JD, Lin SP, Tzen CY, Shen EY, Shih CS
Pediatr Nephrol 2001 Aug;16(8):653-7. doi: 10.1007/s004670100628. PMID: 11519896

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