Success stories

01 Meet the children

02 Meet Irai - Success story

Meet Irai - Success story

Irai is one of the 150 patients affected worldwide by Aromatic Amino Acid Decarboxylase deficiency –AADC deficiency– also known as juvenile Parkinson's. A disease that, until now, had no cure. However, gene therapy is changing the history.

The first birth

Irai was born on the 16th of July 2014. Although it seemed normal during the first weeks, it soon became obvious that something was wrong.

A year after her birth, a diagnosis was made. She suffered from an ultra-rare disease known as juvenile Parkinson's.

This deficiency causes the brain to seem disconnected from the body, keeping it from moving, holding the head up or speaking, among other symptoms.

"Imagine what went through our heads -her father said Irai wouldn’t ever be able to talk, walk, and would probably have to wear a gastric button to allow her to eat... and there was no cure".

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Everyday life

Everyday life with Irai was extremely difficult. She barely slept for three hours. When she was awake, her constant pain caused her permanent wailing.

She suffered crises in which her eyes looked up, stiffened herself and emitted groans that made her great pain evident. Then she was exhausted.

Hopping she would achieve some improvement, they treated her, first, in the Hospital de Barcelona and then in a specialized center in Alicante, where some improvements were obtained, but just it.

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Taken by surprise

The phone rings. A friendly stranger tells them options
they simply cannot believe.

The President of Fundación Columbus, talks to them about gene therapy, the successes they were having with juvenile Parkinson's and -what was most stunning- they found the way to introduce the gene into Irai which could cure her.

On February of 2019 Fundación Columbus informed them that Professor Krystof Bankiewicz, Professor and Director of the Brain Health Department at The Ohio State University and one of the world’s leading experts in juvenile Parkinson's, would treat Irai.

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The second birth

On 14th of May 2019, in Varsovia's Brodno Hospital, Professor Krystof Bankiewicz and his team performanced a highly complex gene therapy surgery.

Definetly it was a success. From then on, Irai's brain would be able to create neurotransmitters that would restore the connection between her brain and her body.

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Gene therapy, Columbus Children's Foundation and Fundación Columbus with the support of Viralgen, are achieving what just a few years ago seemed like a miracle. Now, children and their families, like Irai's one, hope for a "second birth".













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