GENE THERAPY, THE FUTURE OF RARE DISEASES
Yesterday the parents of Guillem and Celia were in a talk about gene therapy in Valencia, organized by Helpify (funding platform for medical research promoted by the CIMA of the University of Navarra). In her, Gloria González, director of the program of gene therapy of the CIMA (Applied Medical Study Centre), explained that this therapy is already a reality, and that it is getting the improvement and cure of rare genetic diseases.
We left this talk very happy and hopeful with the research of Dr. Lipton, who investigates a gene therapy for our children’s syndrome. More than ever we look forward to a future where a treatment for MEF2C is possible. For this research to continue, it is necessary to continue raising funds to finance it, but we are more and more people fighting for this cause and we are convinced that we will succeed.
We have the most important thing: a doctor and an experienced team researching, and the illusion and support of everybody. We continue to fight for our children, because the impossible is just more demanding.
MEF2C Association
We look forward together
