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Bones? Heart? Lungs? How are they related? There is no treatment for the cause of Morquio A. The signs and symptoms that go along with this unpredictable disease may affect multiple organs and systems. Click here to learn more about Morquio A.

what is morquio a?

Morquio A is a rare inherited disease that affects major organ systems in the body. The disease is a form of mucopolysaccharidosis, which is a type of lysosomal storage disorder.

People born with Morquio A can't break down glycosaminoglycans (GAGs) molecules because their bodies don't make enough of an enzyme, or protein, called N-acetylgalactosamine-6 sulfatase (GALNS). This enzyme breaks down or recycles materials the body can't use. When the body doesn't produce enough of the enzyme, GAGs build up in tissues, bones, and major organs. GAGs cause serious problems, including heart disease, skeletal abnormalities, vision and hearing loss, difficulty breathing, and early death.

Even though Morquio A is serious, it's important to remember that the disease can affect people differently. With proper treatment, people with Morquio A can live a long time — in some cases, just as long as people who don't have Morquio A

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cause

Morquio A is a recessive inherited disease. This means that both parents must have a defective gene in order for there to be a risk of passing Morquio A on to their children.

The genes we get from our parents determine most of our physical characteristics. Hair color, blood type, gender, and eye color are all examples of inherited characteristics. But some health problems can also be inherited.

  • In the case of Morquio A, the inherited genes can't make the enzymes, or proteins, that break down a certain type of cellular waste.
  • The waste is called keratan sulfate (KS), and it builds up in cells over time. KS is a type of glycosaminoglycan (GAG).
  • When CAGs build up in the body, the results can be severe. People with Morquio A have widespread, life-limiting physical problems and dangerous organ complications.

TIP/ Exercise

Ask your doctor about the different kinds of low-impact exercises you can start doing to keep yourself strong and healthy. These should be exercises you can safely do at home by yourself or with the help of a caregiver. For some people, aqua therapy brings a combination of movement and relaxation to the muscles that land based exercise can't always provide. See more tips.

Early Signs & Symptoms

The signs and symptoms of Morquio A are not usually obvious at birth. By the age of 2 or 3 years, enough KS has built up in the child's body to start causing problems.

The first noticeable symptoms that usually show are oddly shaped bones, knock knees, spine curvature, and irregular chest growth. As the child gets older, more serious symptoms will start to show up. In some cases, symptoms can show up in children as young as 6 months.

Tests/Diagnosis

Morquio A is a progressive disease, meaning that it will get worse over time. According to the recently published “International Guidelines for the Management and Treatment of Morquio A Syndrome,” it's important to get an early diagnosis in order to prevent further damage, and to start treatment with an enzyme replacement therapy (ERT) right away. The chart below shows the two most common to tests to check for Morquio A.

For the test to screen for Morquio A, Doctors test urine to measure glycosaminoglycan (GAG) levels. People with Morquio A usually have high levels of keratan sulfate, a type of GAG, in their urine. For the test to diagnose Morquio A, Doctors use an enzyme test to measure levels of N-acetylgalactosamine-6 sulfatase (GALNS) in blood and skin cells. People with Morquio A have low amount of this enzyme.

Doctors also perform family testing for Morquio A. Testing is available for parents who already have a child with Morquio A to see if any future children will be at risk. In this prenatal test, fetal cells are collected and the GALNS enzyme is measured. People who have siblings with Morquio A may want to take a genetic test, especially when they are planning their own families. This test shows if they are a carrier of the nonworking gene.

Indication

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 Patient and Physician Support (BPPS) at 1-855-MORQUIO (1-855-667-7846).