Deterioration of the auditory brainstem response in children with type 3 Gaucher disease.
case_series · Level IV
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Abstract
Enzyme replacement therapy (ERT) has improved the quality of life in patients with Gaucher disease (GD). An emerging concern is whether ERT can also halt the neurologic progression in type 3 GD. The authors examined the auditory brainstem response (ABR) in eight children with type 3 GD undergoing high-dose ERT and found a consistent deterioration in ABR response. They conclude that ERT does not halt brainstem degeneration and that alternative therapies must be sought.
Medical subject headings
- Evoked Potentials, Auditory, Brain Stem
- Gaucher Disease