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.

Annabelle’s story

quotes We feel like we’ve hit these little milestones along the way—in her stamina and in her endurance. Which is amazing, because she is now being a kid. And that’s important. quotes

Meet Stephanie, whose daughter Annabelle was diagnosed when she was 4 months old. Now at age 7, Annabelle is on VIMIZIM® (elosulfase alfa), and her mother shares how it has changed the way Annabelle lives with Morquio A.

Learn more about Annabelle’s experience with VIMIZIM.

“My daughter Annabelle started her VIMIZIM infusions in September 2011 when she was 5 years old. Before her first infusion, my husband and I prepared her by telling her that she was going to get an IV in her arm. We were honest with her about what was going to happen and she just went along with it. Annabelle did great.


Since that first infusion, VIMIZIM treatment has become a weekly routine for us. Every Wednesday we wake Annabelle up bright and early to get her dressed and out the door so we can take her to her treatment. We have a 7 am appointment, and we’re there for about 5 hours. It takes up a big part of the day, but it’s absolutely worth it. We are definitely seeing the benefits of VIMIZIM for Annabelle.

During her infusions, Annabelle likes playing games, coloring, listening to music, and watching fun or educational shows on the iPad®.a Sometimes she’ll put the iPad aside and write stories or draw pictures. She also has access to schoolwork that she might be missing because of her treatments. Her teachers have been amazing by rearranging class schedules and making sure she doesn’t fall behind.

We also get a lot of support from the staff at the hospital. They are like our second family. We usually have the same few nurses that work with us to give Annabelle her infusion. We celebrated Annabelle’s birthday in the bed during treatments, and they brought in cupcakes and a clown for her. She has fun with everyone that comes by to say hello to her while we’re there.

I’ve seen a dramatic improvement in Annabelle’s condition since she started treatment. Now she is interested in activities like riding her bike and playing with other kids—things she didn’t do as much before. I can tell that her stamina is getting a whole lot better.”

a iPad is a trademark of Apple, Inc., registered in the U.S. and other countries.


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