Sioux Falls, South Dakota
"Everyone has struggles and troubles in their life, it's how we deal with them that matters," says Kendra, a senior at Augustana College in Sioux Falls, South Dakota.
She should know. Standing just over three feet tall, this giant-spirited young woman has courageously struggled through a life-long medical condition called MPS VI, also known as mucopolysaccharidosis VI or Maroteaux-Lamy Syndrome, which has left her with crippling back problems, bent knees, difficulty walking and breathing, stiff curled hands, and poor eyesight. She sleeps with oxygen at night to keep her organs healthy and moves around her house with a walker. And while she may need a little help getting ready in the morning, tying her shoes, zipping up and closing snaps, this strong, intelligent, effervescent young lady has more horsepower than the motorized chair she uses to zip around town.*
"I may be small, but I have a big attitude!" she says.
"Inside, I use a walker. I do not bring my power chair in the house. I use para-transit to get to school and back, and I'm fortunate that my campus is pretty handicapped-accessible--they have push buttons for doors, which is really helpful."
Kendra was diagnosed with MPS VI at age four and has been receiving Naglazyme infusions for six years.
"I've received about 343 treatments," she recalls.
The Long road to diagnosis
Kendra's symptoms started early but it took several years for doctors to accurately diagnose her condition. Since then she's endured numerous surgeries starting when she was just over one year old. Before learning to walk, doctors had her on the operating table to correct a ‘gibbus' or a bump on her back, which is a common characteristic of patients with MPS VI.
"My mother first noticed I had a bump on the lower part of my back when I was six-months-old. The local doctor watched it for a few months and then sent us to an orthopedic doctor. He decided that surgery to fuse my back would stop the bump from progressing and give me a better chance for my back to grow straight. This was before my diagnosis."
By age 16 she was back in the hospital for spinal decompression surgery.
"My condition is very rare and at that time in 1985, I don't think the doctor was familiar with MPS VI. It ended up after that operation, and then after my diagnosis, we weren't sure if I ever really needed that surgery."
For several years following her first surgery, Kendra continued follow-up visits with her orthopedic doctor, who, after much confusion and difficulty identifying the actual cause of her back problems, finally referred her to a geneticist.
"The geneticist knew right away that there was something wrong and began chromosome tests and concluded with the diagnosis of MPS VI at the age of four."
"My biggest difficulty with having MPS is the question of will I have or need more surgeries? Going into the OR is very scary. My advice with surgery is talk to your doctors, speak up if you have concerns, they will listen to you. I asked lot of questions and told them to explain things as simply and clearly as possible."
Kendra is an active participant in every aspect of her medical treatment and has a strong conviction about using her personal experience and knowledge to help others, which is why she's pursuing a double major in psychology and sociology.
"Having MPS VI has affected my life in many ways and has guided my career choice. I would like to counsel children and their families in a hospital setting. I've had wonderful people help me throughout my tough times and I want to be there for others during their tough times."
"My goal in life is to help others. I love to laugh and have fun with friends and family. I may seem shy, but once you get to know me you'll find out I'm not. I am more like everyone than different. I may be small, but the only part of me that is small is my size. My brain and heart are as big as they come."
"I hope no one feels sorry for me because I do not. I have a happy life. I am capable, bright and determined to reach all the goals that I set out for myself."
Kendra's favorite quote: "When life hands you lemons, make lemonade.
* Individual patient response to Naglazyme® treatment will vary.
Full Indication and Important Safety Information
Naglazyme® (galsulfase) is indicated for patients with mucopolysaccharidosis VI (MPS VI). Naglazyme has been shown to improve walking and stair-climbing capacity.
Important Safety Information
The most common adverse events in patients treated with Naglazyme were headache, fever, joint pain, vomiting, upper respiratory infections, abdominal pain, diarrhea, ear pain, cough, and ear infections. Severe reactions included swollen blood vessels, low blood pressure, difficulty breathing, respiratory distress, stopping breathing, and hives.
The most common symptoms of infusion reactions included fever, chills/shakes, headache, rash, and mild to moderate hives. Nausea, vomiting, elevated blood pressure, chest pain, abdominal pain, malaise, and joint pain were also reported.
No patients discontinued Naglazyme treatment because of reactions. Nearly all patients developed antibodies as a result of the treatment, but the level of immune response did not correlate with the severity of the adverse reaction.
Because antihistamine use may increase the risk of stopping breathing, airways should be checked to ensure they are not blocked or obstructed. Treatment may be delayed if you have a fever or respiratory illness..
To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Naglazyme is available by prescription only. To learn more, please visit www.naglazyme.com for full prescribing information. If you have any questions about this information, please talk with your doctor.