Gene Therapy
bluebird bio, a company located in Boston, MA has conducted clinical trials for ALD gene therapy. Results have been published (see below) and gene therapy treatment is promising to be an option for treatment for cerebral ALD. Gene therapy for ALD has yet to go through FDA approval. If approved, it will be a treatment option for children with cerebral ALD that offers an alternative to allogeneic hematopoietic stem cell transplant, eliminating the need for a bone marrow match.
For more information:
bluebird bio: [email protected]