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mucopolysaccharidosis type 4B

MIM.253010 3p21.33

Autosomal recessive Morquio syndrome B, also referred to as mucopolysaccharidosis type IVB (MPS4B), is caused by mutation in the gene encoding beta-galactosidase (GLB1) (MIM.611458).

Morquio syndrome B is allelic to the various forms of GM1-gangliosidosis (MIM.230500). The latter disorders show central nervous system involvement.

Synopsis

- adult height 82-115 cm
- short-trunked dwarfism
- mildly coarse facial features
- prognathism
- hearing loss
- corneal opacities
- broad mouth
- widely spaced teeth
- grayish enamel
- frequent caries
- valvular heart disease Aortic valve stenosis
- intimal thickening in the coronary arteries
- frequent upper respiratory tract infections
- restrictive lung disease
- flaring of rib cage
- prominent sternum
- inguinal hernia
- mild hepatomegaly
- osteoporosis
- platyspondyly
- odontoid hypoplasia
- cervical subluxation
- kyphosis
- hyperlordosis
- scoliosis
- ovoid vertebral bodies
- coxa valga
- constricted iliac wings
- joint laxity
- genu valgum
- ulnar deviation of the wrist
- epiphyseal deformities of tubular bones
- widened metaphyses
- pointed proximal second through fifth metacarpals
- normal intelligence
- cervical myelopathy
- beta-galactosidase deficiency in fibroblasts and white blood cells
- keratan sulfate excretion in urine with age
- appear normal at birth
- onset between 1-3 years

References

- Gucev ZS, Tasic V, Jancevska A, Zafirovski G, Kremensky I, Sinigerska I, Nanba E, Higaki K, Gucev F, Suzuki Y. Novel beta-galactosidase gene mutation p.W273R in a woman with mucopolysaccharidosis type IVB (Morquio B) and lack of response to in vitro chaperone treatment of her skin fibroblasts. Am J Med Genet A. 2008 Jun 10. PMID: 18546276