2 doctors weighed in:

What is effective therapy for gaucher disease?

2 doctors weighed in
Dr. Johanna Fricke
Pediatrics - Developmental & Behavioral
1 doctor agrees

In brief: Adults & children

with GD Type 1 may have symptom reduction or reversal from Enzyme Replacement Therapy.
Different ERT's are approved for kids & adults. Substrate Reduction Therapy is given to adults with mild Type 1 who can't take ERT. No treatment can reverse brain damage in patients with Type 2 or Type 3. Mutations of a gene on Chr. 1p22 cause all 3 Types. Call the National Gaucher Foundation, 800-504-3189.

In brief: Adults & children

with GD Type 1 may have symptom reduction or reversal from Enzyme Replacement Therapy.
Different ERT's are approved for kids & adults. Substrate Reduction Therapy is given to adults with mild Type 1 who can't take ERT. No treatment can reverse brain damage in patients with Type 2 or Type 3. Mutations of a gene on Chr. 1p22 cause all 3 Types. Call the National Gaucher Foundation, 800-504-3189.
Dr. Johanna Fricke
Dr. Johanna Fricke
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