The first drugs capable of repairing DNA are here! Gene therapy is no longer a dream, it has become a reality for patients. In 2019, the Telethon celebrates this medical revolution with a fierce determination to multiply Victories, so that one day each patient has the “chance” to receive treatment.
These first major therapeutic victories for rare diseases reflect the determination of families, researchers, and the incredible mobilization of donors, volunteers and partners of the Telethon for 30 years.
We have truly entered into a new era for medicine, which enables to stop and conquer diseases previously considered incurable. This is an unprecedented medical revolution, and gene therapy, which we have supported sometimes against all odds, is its strongest link.
Today, we are proud and moved when thinking about the thousands of patients who finally benefit from a clinical trial when, for so many years, there was nothing for these diseases with a relentless progression.
Our major victories finally bear the name of medicines and save lives.
Because overcoming the disease is possible, let us multiply Victories for all those who await!
These victories, it is the families that speak the best of them.
“This is what I have been waiting for 10 years. There is finally a treatment for my son’s disease. Here we are. Thank you to the doctors, the researchers, and the Telethon!”
Says Géraldine, very emotional, when speaking about the treatment that her son Lee is expected to receive shortly.
Chonthicha’s face lights up when speaking about the gene therapy she benefited from and which freed her from the blood transfusions imposed by her illness, beta-thalassemia:
“I have not had a transfusion for the past three years, while I used to have them every month. It is a new life. I am lucky!”
Says the young woman.
For Laetitia and Julien, the spinal muscular atrophy diagnosis of Oscar, at just one-year old, is a terrible shock. They pin all their hopes for their little boy on research. For the past year and a half, Oscar has benefited from the first available treatment.
“This is revolutionary for us. It changed our life. Thanks to research!”
Praises his mother.
After three years of hospitalizations, transfusions, and constant monitoring, the life of Sethi, this little boy with blonde locks suffering from a very rare immune deficiency, is no longer in danger.
“Sethi can run, go to school. Gene therapy has offered a new life to my son!”
What a great pleasure for Azizah, his mother.
“These images… as a mother, I dreamt of for so many years”.
This mother, moved to tears, is Laurence Tiennot-Herment, the President of AFM-Telethon. Like many parents who have come together in the association for 60 years, she pursues one aim: finding a cure!