Enzyme replacement therapy is available to treat mainly Fabry disease and Gaucher disease, and people with these types of sphingolipidoses may live well into adulthood. The other types are generally fatal by age 1 to 5 years for infantile forms, but progression may be mild for juvenile- or adult-onset forms.
Enzyme replacement therapy is available for treating mainly Fabry disease and Gaucher disease, allowing people with these sphingolipidoses to live into adulthood. The other types are usually fatal by age 1 to 5 years in infantile cases but may progress mildly in juvenile or adult-onset forms.