“What is the best treatment for Gaucher’s disease?”
Our 12 year old daughter has Gaucher’s disease. What is the best treatment for Gaucher’s disease?
5 Answers
There are different approach depending on the type. For example, Types 1 and 3 respond well to Enzyme replacement therapy. Cerezyme (Imiglucerase) by Sanofi Genzyme, VPRIV by Shire, and Elelyso (Talyglucerase) by Pfizer are USDA approved enzyme replacement. Regular physical exam is imperative. Other options are bone marrow transplant, blood transfusion, spleen surgical procedure and medicines. Thank you.
You need to ask your pediatrician to refer you to a specialist in genetic diseases such as the Genetic center in Johns Hopkins Hospital or Gauchersdisease.org
Gaucher's disease is typically treated by Metabolism doctors. There are a few treatment options, including enzyme replacement therapy and bone marrow transplantation.
You need to ask the doctor treating her. If you have questions about that, get a second opinion. This is not my area of expertise.
RB Thomas, MD
RB Thomas, MD
Cerezyme is the specific enzyme to replace what is lacking. This must be given every 2 or 3 weeks by infusion. There is also an oral drug that may be able to be used instead in some patients depending on the severity of disease and how they respond to it.
