Juvenile onset acid maltase deficiency presenting as a rigid spine syndrome

Neuromuscul Disord. 2006 Apr;16(4):282-5. doi: 10.1016/j.nmd.2006.02.001. Epub 2006 Mar 13.

Abstract

The rigid spine syndrome is a disorder characterized by proximal muscle weakness and limitation in flexion of the cervical and dorsolumbar spine. Such phenotype may be caused by a variety of hereditary myopathies. We present the case of a 15-years-old boy with rigid spine syndrome and severe restrictive respiratory changes. Muscle biopsy revealed vacuolar myopathy with excessive deposition of PAS-positive material. Lysosomal acid maltase activity in cultured skin fibroblasts was reduced to 6% of control values. DNA analysis demonstrated novel mutation E888X of acid alpha-glucosidase gene with compound heterozygosity IVS1/E888X, confirming diagnosis of Pompe disease. We conclude that acid maltase deficiency should be considered in the diagnosis of rigid spine syndrome.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Biopsy
  • Glycogen Storage Disease Type II / complications
  • Glycogen Storage Disease Type II / diagnosis*
  • Glycogen Storage Disease Type II / genetics
  • Humans
  • Male
  • Muscle Weakness
  • Muscles / pathology
  • Mutation
  • Spinal Diseases / diagnosis
  • Spinal Diseases / etiology*
  • Spinal Diseases / genetics
  • Spine / abnormalities
  • Syndrome
  • alpha-Glucosidases / genetics*

Substances

  • GAA protein, human
  • alpha-Glucosidases