A member asked:

Please advice about the following therapy for thalassemia purpose bluebird bio gene therapy hydroxyurea therapy when the therapy will be available?

A doctor has provided 1 answer
Dr. David Masiello answered

Specializes in Hematology and Oncology

Now: Hydroxurea is already available. I believe what you are inquiring about is gene therapy, inserting the normal beta globulin gene into someone with a hemoglobinopathy. This is currently in development and it is unclear when this, if ever, will be available outside a clinical trial.

Answered 4/16/2017

3.7k views

Thank

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