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Ehlers-Danlos syndrome progeroid type

MedGen UID:
1841549
Concept ID:
CN030853
Disease or Syndrome
Synonym: Ehlers-Danlos syndrome, spondylodysplastic type
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).
 
Related genes: B3GALT6, SLC39A13, B4GALT7
 
Monarch Initiative: MONDO:0007526
Orphanet: ORPHA75496

Definition

A form of spondylodysplastic Ehlers-Danlos syndrome due to variants in <i>B4GALT7</i> and characterized by short stature, variable degrees of muscle hypotonia, joint hypermobility, especially of the hands, and bowing of limbs. Additional features include the typical craniofacial gestalt (mid-face hypoplasia, round, flat face, proptosis and narrow mouth), hyperextensible skin that is soft, thin, translucent and doughy, delayed motor and/or cognitive development, characteristic radiographic findings (such as radio-ulnar synostosis, radial head subluxation or dislocation, metaphyseal flaring and osteopenia) and ocular abnormalities. [from ORDO]

Recent clinical studies

Diagnosis

Sellars EA, Bosanko KA, Lepard T, Garnica A, Schaefer GB
Semin Pediatr Neurol 2014 Jun;21(2):84-7. Epub 2014 Apr 13 doi: 10.1016/j.spen.2014.04.007. PMID: 25149931

Prognosis

Sellars EA, Bosanko KA, Lepard T, Garnica A, Schaefer GB
Semin Pediatr Neurol 2014 Jun;21(2):84-7. Epub 2014 Apr 13 doi: 10.1016/j.spen.2014.04.007. PMID: 25149931

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