Innovative therapeutics



Morquio A Syndrome (MPS IVA) Patient
Alexandria, Virginia

Annabelle - BioMarin Patient StoriesStephanie and her husband Austin have spent years in a desperate chase to find a treatment to save their daughter’s life and alleviate the crippling effects of Morquio A (MPS IVA). This rare genetic disorder, also known as MPS IVA, causes skeletal dysplasia, spinal malformation, heart and respiratory failure and other severe organ damage. Since her diagnosis at 6 months, Annabelle has braved countless doctors’ visits, invasive surgery and a lifetime of pain. Others like her have been fighting the disease for years. 

“Every minute of every day counts for us. Because every day that goes by without a treatment, we know that MPS IVA is taking its toll on our daughter’s body,” says Stephanie, Annabelle’s mother. “We are literally in a race against time to get ahead of the damage caused by this disease, and as parents, we cannot watch this happen without a fight.”

Annabelle participated in the clinical trials for Vimizim® (elosulfase alfa), which won approval from the U.S. Food and Drug Administration for the treatment of MPS IVA.

“I’ve seen a dramatic improvement in Annabelle’s condition since she started treatment,” says Annabelle’s mother. “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.”

Important Safety Information

Life-threatening allergic reactions, known as anaphylaxis, can occur during Vimizim 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, taking any medication, or 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 RareConnections at 1-866-906-6100.

Please see accompanying full Prescribing Information by going to, including important warning.

Read our patient stories…



Kendra - BioMarin Patient Stories

Watch our patient videos…

Kendra - video

Annabelle - video



Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU



Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

Global Medical Information

Contact Global Medical Information