WARNING: Life-threatening allergic reactions have occurred in some patients during VIMIZIM® (elosulfase alfa) infusions and up to 3 hours after infusion. Patients with acute respiratory illness may be at increased risk and require additional monitoring.

Sarah’s story

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Meet Sarah, a 16 year old living with Morquio A. She and her mother Ruth share their journey, which began when Sarah was diagnosed at 2 and a half years old and continues today, as she receives her weekly treatment with VIMIZIM® (elosulfase alfa).

Learn about Sarah’s experience with her weekly VIMIZIM infusions.

“I had concerns before I started VIMIZIM infusions, mostly because I’m a huge needle-phobe. The second I heard it was an infusion, I got really scared. I wanted to know if there was anything they could do to lessen the pain of the needle. I ended up using a port and I really like it. It helps with the needle fear. The nurses put numbing cream on it and they can also use it to draw blood, which makes things so much easier for me. I’m happy I got the port. It’s just worth it.


And let me add that my nurses are absolutely amazing, and I’ve been with them for a year and a half, so we totally understand each other. There are some nurses I know better than others, but they’re all very accommodating.

I try to keep busy on my infusion days. Since I don’t go to school on those days, I bring homework to do at the infusion clinic. When I first started, I didn’t know how to use my time. I showed up with tons of homework. I didn’t realize how tired I’d get during the infusion (because they give me an antihistamine to help prevent a reaction) so I didn’t get to all of that homework.

My advice is to not bring tons of things that you expect to do, because you can get distracted by the activity of the room, and you may get sleepy. Usually I’ll do a few hours of work and then I’ll watch movies, or just read for a while. If it’s summer, I’ll jump on Facebook and catch up with my friends. I’ve got about 4 to 5 hours to fill.

I wish I’d known to talk to my teachers before I started getting infusions. They all understand now, but it took some explaining to make sure they got how important these infusions are to me and my health. Having those conversations really helped. And making sure to get the work I’m going to miss because of my infusion day helps, too!

I’ve missed a few infusion sessions because of vacations, or midterms and finals, but I try to reschedule if I can. My energy is up. And I’m able to do more of the things I want to do that I couldn’t before because I’d get tired. The treatments can take a long time, and there can be side effects, but it’s so worth it.”

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VIMIZIM® (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

Important Safety Information

Life-threatening allergic reactions, known as anaphylaxis, can occur during VIMIZIM® (elosulfase alfa) infusions. Typical signs of anaphylaxis include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. Contact your doctor or get medical help right away if these symptoms occur during or after VIMIZIM infusions. If you have a respiratory illness, you may be at risk for a sudden worsening of your condition, and you may require additional monitoring.

VIMIZIM is a prescription medicine. Before treatment with VIMIZIM, it is important to discuss your medical history with your doctor. Tell your doctor if you are sick or taking any medication and if you are allergic to any medicines. Also tell your doctor if you are pregnant, planning to become pregnant, or are a nursing mother. Your doctor will decide if VIMIZIM is right for you. If you have questions or would like more information about VIMIZIM, contact your doctor.

Anaphylaxis can occur during any VIMIZIM infusion and up to three hours after any infusion, and hypersensitivity reactions have been observed as early as 30 minutes from the start of infusion but as late as six days after infusion.

Serious and severe reactions can happen with VIMIZIM treatment, including life-threatening allergic reactions (anaphylaxis), hives, swelling, cough, shortness of breath, and flushing. You should receive medication such as antihistamines before VIMIZIM infusions to reduce the risk of reactions. If a reaction occurs, the infusion should be slowed or stopped and you may be given additional medication. If a severe reaction occurs, the infusion should be stopped immediately and you will receive appropriate medical treatment.

If you have acute febrile or respiratory illness at the time of VIMIZIM infusion you may be at higher risk of life-threatening complications from hypersensitivity reactions. If you use supplemental oxygen or continuous positive airway pressure (CPAP) you should have it available during your infusion in the event of a sudden reaction, or extreme drowsiness/sleep from antihistamines.

Spinal cord damage may occur due to the natural MPS IVA disease process. Signs of spinal cord injury include back pain, numbness and paralysis, and loss of bladder and bowel control. Contact your doctor immediately if you develop any of these symptoms.

The most common side effects reported during VIMIZIM infusions included fever, vomiting, headache, nausea, abdominal pain, chills, and fatigue. These are not all of the possible side effects with VIMIZIM. Talk to your doctor if you have any symptoms that bother you or that do not go away.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

For more information, call BioMarin RareConnections™ at 1-866-906-6100.


BioMarin Pharmaceutical Inc.

Novato, CA 94949

BioMarin RareConnections™: 1-866-906-6100