What experts say

Ritva Tikkanen, PhD, is a professor of biochemistry and molecular biology at the medical faculty of the University of Giessen in Germany. She has been involved in research on AGU for almost thirty years, since starting her Master Thesis in Finland. Dr. Tikkanen shared her perspective with us:

“Patients and families confronted with the diagnosis for a rare disease often face many obstacles like lack of information about the disease, and especially lack of any kind of therapies. There is a tremendous need for novel therapies for rare diseases, including AGU. I have been following the research field for AGU since almost 30 years now, and finally, there is a sparkle of hope for all the AGU patients and their families. I am extremely happy to know that a therapy might soon be available for patients suffering from this devastating disease. I would like to strongly support this important project that will help to save the lives of a number of wonderful kids like Makeda. Every donation, no matter how small, is a further step towards the goal, gene therapy for AGU!”

View the latest research paper on gene therapy for AGU, co-authored by Dr. Tikkanen – Pre-clinical Gene Therapy in AGU.