Los Angeles, California
"I've been many places and I've done many things, but nothing can compare to being in the Naglazyme® clinical trials in 2002. There were eight of us participating in the study and I think we made history. Because of that, now there's help for everyone with MPS VI," Shafon proudly recalls.
A high school senior from Los Angeles, Shafon is one of those extraordinary individuals who can see only the silver lining and never the clouds in life.
"It was such a blessing for me because my body has changed a lot by participating in the study and taking Naglazyme."
Despite her inspiring enthusiasm, Shafon can only speak with great effort because she has a tracheotomy and must use a mobile voice box to communicate verbally. If you were judging by her demeanor alone, however, you would never imagine that she experiences very much discomfort of any kind. By the looks of it, she's a sassy fashion diva with a radiant smile and a zest-for-life to match.
"When I was born my mom said I appeared to be a healthy and normal baby girl. Unfortunately, I was not. When I was one-year-old I began to have chronic ear infections and colds all the time. I was always in and out of the doctor's office and became immune to antibiotics. This went on for about another year," reports Shafon.
"At the age of two my mom, dad and my Aunt Viv," she continues, referring to her Aunt Vivian with whom she lives today, "decided to take me to the children's hospital to see what was going on. We stayed all day and all night long and they did a lot of tests; a lot of doctors came in and out. First they came up with the wrong diagnosis but then we got a second opinion of MPS VI."
"But I haven't really been sick a lot," recalls Shafon, who has a remarkable ability to take her serious health problems in stride.
"In 2006 I had open heart surgery which was a success and I've also had cornea transplants. But mostly I have the regular stuff that comes with MPS VI like short stature, wide nose, sore throat, chest pains, ear problems, breathing difficulties; enlarged tongue, hands, feet liver and spleen; and some bone and joint problems."
"Since she's been on the enzymes her little joints have stayed real stiff but they used to wouldn't move at all. Now we can move them some since she's been taking Naglazyme," reports Aunt Vivian.*
"I am a very blessed child," she says. "I have my own charitable foundation and I've brought together a lot of people with MPS VI."
Shafon's physical disabilities do not appear to slow her down. Not even blindness.
"At first she wanted to be a lawyer. She used to tell her mom that she wanted to go to college to be a lawyer, but now she wants to be movie star!" adds Aunt Vivian.
"She does all kinds of things—special things!—she worked at Wal-Mart, and they were so glad to have her because she could manage all the bottom shelves! So that way she saves people's knees! --You know, when we get older our knees get to hurting us anyway!"
"She's worked at the nursery and a fabric store where she used to take care of all the thread and buttons. She's done that and she's volunteered at a place for handicapped children where they taught her how to do a little filing and stuff like that. She can also write and use the computer very well," says Vivian, who barely takes a breath to continue her niece's long list of accomplishments…
"When people haven't met her or don't know her, especially younger children, they kind of get afraid of her a little bit because of how she looks; some kids run away. But older people love her, when they first meet they see she's fun and outgoing. She has lots of friends and usually everybody accepts her for who she is."
Undaunted by what others may consider intolerable disabilities, Shafon is only slightly inconvenienced by her mobility problems.
"But now she has a scooter and since her open heart surgery she's breathing better, so she can walk more like her doctor orders. She tells her doctor, she says, ‘I'm making my five laps a day!'" continues Aunt Vivian.
"She walks five laps a day. So she's getting better!"
"Oh! And dancing, too! She wants to dance with her doctor!..."
* 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.