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Babies with
lysosomal storage disorders usually appear normal at birth. Problems
progressively appear in the first few years. These may include:
- Changes in
the facial appearance bone
deformities and joint stiffness
- loss of skills,
such as speech and learning
- behaviour
problems, and mental retardation
- sight and
hearing difficulties
- frequent
respiratory infection and heart disease
- enlarged
organs, such as liver and spleen
Disease severity
is variable. Some patients may survive into adulthood, but patients
who are more severely affected die in their mid-teens or earlier |
