The families testify

Today, Angel is doing well

For Susie and José, the parents of Angel, all began the day when Angel’s older brother was diagnosed with adrenoleukodystrophy. Angel is 4 years old. The parents are overwhelmed. Especially because there is a risk Angel carries the same gene. The results are issued. The child bears the same disease. With their two sons sick, Susie and José get in touch with the ELA office in Spain. Unfortunately, the disease progresses quickly, too quickly. The condition of the elder brother deteriorates and the disease takes him away 14 months later.

Susie and José will do anything for Angel. Following ELA’s advices, they contact Pr. Patrick Aubourg. The first examinations are conducted. Everything goes very fast and Angel becomes a candidate for the ALD gene therapy. The parents do not hesitate, their kid must participate to this trial. They stay three months in Paris. It was on January 2007. Today, Angel is doing well. The graft halted the disease. He keeps some damages on the visual field, sense of orientation and immediate memory. His parents are pleased because it was clear the disease would have progressed without gene therapy like it happened for his brother.

 

Andrès was diagnosed on time

” Before we were a normal family ” says the older brother of Andrès, the most recent little boy treated by Pr. Aubourg. His adrenoleukodystrophy was diagnosed fortuitously. The child is admitted to the hospital because of an important gastroenteritis. The standard analysis show important abnormalities. At first, physicians think the results are erroneous and decide further investigations which will lead to the final diagnosis of adrenoleukodystrophy.

They met with several neurologists who explained that if the disease progresses, Andrès will only have 2 years to live, that he will start losing his vision, hearing and all his vital functions. The parents are overwhelmed. Following the advices of their neurologist, they travel to Paris for an appointment with Patrick Aubourg. Andrès will be the third transplanted child. These are very difficult times between examinations, transplantation and travels between Spain and France. The mother says that she “never saw him sick because he was diagnosed on time”. Andrès’ parents can take a breath, they are still afraid but so far the examinations are good.