A Bright Future for Kaya

When my son Kaya was born, I knew deep down inside that something was wrong. The doctors, however, waved it away and said I was just worrying since our oldest daughter had passed away due to a congenital heart defect. As Kaya grew older we began to notice differences. A friend of mine pointed out that his crawling was unusual and suggested I have him seen by a doctor. At the age of three, doctors discovered Kaya had muscular dystrophy. We were devastated and desperate not to lose another child. It took almost a year to find out that Kaya had Limb Girdle Muscular Dystrophy type 2D (LGMD2D). LGMD2D causes weakness of the muscles in the hip, shoulder, and abdomen. The severity of the disease varies greatly between individuals.

The day Kaya was diagnosed, I was urged to act and find a way to save my child. I was ready to do anything for my little boy and would go to the end of the world if I had to. Living in the Netherlands left us with very few options. There were only 11 other children diagnosed with LGMD2D in the Netherlands, with research money going towards diagnoses with the largest patient populations, We were told there was no treatment and that my son would be in an electric wheelchair by 10 years old. I had to find something. It was a matter of life—my son’s life. Every evening when my kids went to bed, I would search the internet trying to find information and a cure for my son.

Finally, what we were hoping for, for so many years, happened. The summer of 2015, we received a phone call from Nationwide Children’s Hospital. Kaya was eligible to participate in a trial. We were so excited and finally had hope for a brighter future. The day we signed the consent forms was a bit scary as we realized there were some risks and like any other trial, uncertainty. We found reassurance as Dr. Jerry Mendell and the rest of the team answered all of our questions and concerns and ultimately shared the same goal: to find a cure.

Kaya received his first gene transfer therapy with a low dose. Quickly after the gene transfer, Kaya felt some muscles he had never felt before. I will always remember the fortune cookie we had with our dinner that night which read, “You will soon gain something you have always wanted.” It could not be a coincidence; Kaya’s life was changing for the better. His energy increased as well as his quality of life. While he used to rely on a wheelchair to get around, Kaya is now walking and can even climb trees.

More recently, we visited Nationwide Children’s Hospital last February for objective physical therapy tests. Kaya’s muscles continue to gain strength and he has more energy to do the things he enjoys, such as fencing and playing the guitar. He attended the Eurodis Summer School and is now a Certified Patient Expert. Kaya, now 18 years old, is graduating this year and wants to follow Dr. Mendell. He is determined to study biomedical science and give back to the neuromuscular field.

This entire experience not only made Kaya’s muscles stronger, it also made him a much stronger person. I came to Columbus with a young boy who was told nothing could be done. Now, I have a young adult who has changed both physically and mentally. The doctors have not only taken care of his muscle disease, but also his total being. We still hope and believe the other muscles will be treated in time. Even if Kaya stays the way he is, we are more than satisfied and very grateful to be part of a trial that is working to find a cure and eventually treat all muscles.

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When my son Kaya was born, I knew deep down inside that something was wrong. The doctors, however, waved it away and said I was just worrying since our oldest daughter had passed away due to a congenital heart defect. As Kaya grew older we began to notice differences. A friend of mine pointed out that his crawling was unusual and suggested I have him seen by a doctor. At the age of three, doctors discovered Kaya had muscular dystrophy. We were devastated and desperate not to lose another child. It took almost a year to find out that Kaya had Limb Girdle Muscular Dystrophy type 2D (LGMD2D). LGMD2D causes weakness of the muscles in the hip, shoulder, and abdomen. The severity of the disease varies greatly between individuals.

The day Kaya was diagnosed, I was urged to act and find a way to save my child. I was ready to do anything for my little boy and would go to the end of the world if I had to. Living in the Netherlands left us with very few options. There were only 11 other children diagnosed with LGMD2D in the Netherlands, with research money going towards diagnoses with the largest patient populations, We were told there was no treatment and that my son would be in an electric wheelchair by 10 years old. I had to find something. It was a matter of life—my son’s life. Every evening when my kids went to bed, I would search the internet trying to find information and a cure for my son.

Finally, what we were hoping for, for so many years, happened. The summer of 2015, we received a phone call from Nationwide Children’s Hospital. Kaya was eligible to participate in a trial. We were so excited and finally had hope for a brighter future. The day we signed the consent forms was a bit scary as we realized there were some risks and like any other trial, uncertainty. We found reassurance as Dr. Jerry Mendell and the rest of the team answered all of our questions and concerns and ultimately shared the same goal: to find a cure.

Kaya received his first gene transfer therapy with a low dose. Quickly after the gene transfer, Kaya felt some muscles he had never felt before. I will always remember the fortune cookie we had with our dinner that night which read, “You will soon gain something you have always wanted.” It could not be a coincidence; Kaya’s life was changing for the better. His energy increased as well as his quality of life. While he used to rely on a wheelchair to get around, Kaya is now walking and can even climb trees.

More recently, we visited Nationwide Children’s Hospital last February for objective physical therapy tests. Kaya’s muscles continue to gain strength and he has more energy to do the things he enjoys, such as fencing and playing the guitar. He attended the Eurodis Summer School and is now a Certified Patient Expert. Kaya, now 18 years old, is graduating this year and wants to follow Dr. Mendell. He is determined to study biomedical science and give back to the neuromuscular field.

This entire experience not only made Kaya’s muscles stronger, it also made him a much stronger person. I came to Columbus with a young boy who was told nothing could be done. Now, I have a young adult who has changed both physically and mentally. The doctors have not only taken care of his muscle disease, but also his total being. We still hope and believe the other muscles will be treated in time. Even if Kaya stays the way he is, we are more than satisfied and very grateful to be part of a trial that is working to find a cure and eventually treat all muscles.

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