On October 20, 2018, the annual meeting of the French Crigler-Najjar (CN) patients association (Association Française de Crigler Najjar; AFCN) took place at Hôpital Béclère in Clamart, where all the French CN patients are followed.
The families come from all over France to attend the annual general assembly, to meet other families, exchange their experience and hear the latest news about the research on their disease. This year, they especially looked forward to finding out more about the beginning of the new gene therapy trial.
The first part of the meeting was therefore dedicated to the project CureCN.
Fanny Collaud from CureCN coordinator Genethon presented the progress of the project and gave the latest updates. She particularly talked about the challenges facing viral vector administration and mentioned the possibility of re-administrating the vector and also of overcoming the problem with anti-vector antibodies. Aurélie Hubert and François Petit from Hôpital Béclère Clamart were present to answer questions about the trial itself: the enrolment of the patients and the follow-up with the patients before, during and after the injection. Finally, AFCN’s role in the project and the importance of the CN World Registry were explained.
These presentations were very interactive. The families were extremely interested and asked a lot of questions. Overall, the feedback was positive. "We are very happy because everything was explained to us in simple words that enabled us to understand even complicated scientific things," said one mother. Another was excited: “I was concerned about the trial, but now that I know how the trial will be executed, I am very eager that my son can get enrolled."
In general, everybody was exceedingly impressed by the work that has already been done and thankful for all the people engaged in this project.
While CureCN is still at a very early stage and there is still a long way to go, the fact that scientists are investing great efforts in the research of this ultra-rare disease gives hope to Crigler Najjar patients and their families.