A member asked:

How do i know when will gene therapy become a reality for orphan diseases (single gene disorders)?

A doctor has provided 1 answer
Dr. Peter Francis answered

Specializes in Ophthalmology

Glybera: The first gene therapy has now been approved in europe for an orphan disease. Several hundred therapies are currently in human clinical trials. So, i would say gene therapy is now a reality and the next 5 years will see a number of therapies approved.

Answered 7/5/2013

5.2k views

Thank

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