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Mom launches company to cure daughter's fatal disorder

Kevin Chupka
Executive Producer/Writer

If your child was sick you would do everything in your power to make them feel better, right? Well, when Karen Aiach’s daughter, Ornella, was diagnosed with Sanfilippo Syndrome she went above and beyond what most parents would do.

Aiach explains the disease as “an awful neurological disease where the child will apparently develop normally up to a certain age, the age of two, and from that age start regressing from a cognitive standpoint and start entering into severe behavioral disorders.”

She adds the disease is fatal and those affected seldom see their 20th birthday.

Karen Aiach and her daughter Ornella who was diagnosed with Sanfilippo Syndrom in 2009.

So when her daughter was diagnosed with Sanfilippo, Aiach did what any parent would do, which is to try and fix it. While many would try in vain to find a cure, Aiach assembled a group of experts and created a company whose sole purpose was to end the horrible disease.

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Lysogene was started in response to my daughter being diagnosed with that disease,” she says. “Facing the impossible and the unbelievable, I tried to react in a positive way and started discussing and meeting with researchers to find potential solutions to treat that disease and we eventually came altogether with a very promising approach which is gene therapy.”

Gene therapy is a process in which doctors introduce a kind of virus into the patient that carries specialized genes meant to replace missing or broken genes in the cells of a given patient.

Lysogene has completed what is called a “Phase I/II trial” in France with promising results that prove the therapy is safe and show encouraging signs of efficacy in treating the disease.

The next step is to start a Phase II/III clinical trial of its treatment beginning in 2016, using what is called LYS-SAF302 gene therapy. The trials will be conducted both in the United States and Europe. Pre-clinical trials used in Sanfilippo-infected mice show the potential to stop the course of the disease and extend their lifespan close to normal.

If all goes well Lysogene hopes for market approval for the therapy sometime in 2019-2020.

Aiach is content knowing her work could one day help thousands of children afflicted with the condition. As for Ornella, she says, “We have done the maximum that we could do and so we are all very proud of that, I think. And when Ornella looks at me I’m sure she has a tremendous amount of love and recognition because thanks to that experimental treatment we’ve helped her to live better and to have a better quality of life.”

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