Get the details on Morquio A

What is Morquio A?

Morquio A is a form of mucopolysaccharidosis (MPS), a type of lysosomal storage disorder.  Also known as MPS IVA, Morquio A is a rare and progressive inherited disease that affects major organ systems in the body.

What causes Morquio A?

Enzymes are proteins that perform  specific jobs in your body. People with Morquio A do not make enough of a specific enzyme, called N-acetylgalactosamine-6 sulfatase, or GALNS (gal‧en‧es), which breaks down and recycles cellular waste called glycosaminoglycans (GAGs).

When the body doesn't produce enough of the enzyme, GAGs build up and can potentially cause serious physical problems in

  • Tissues
  • Bones
  • Major organs
Healthy cell with a regular amount of GAGs

What is Morquio A?

Morquio A is a form of mucopolysaccharidosis (MPS), a type of lysosomal storage disorder.  Also known as MPS IVA, Morquio A is a rare and progressive inherited disease that affects major organ systems in the body.

What causes Morquio A?

Enzymes are proteins that perform  specific jobs in your body. People with Morquio A do not make enough of a specific enzyme, called N-acetylgalactosamine-6 sulfatase, or GALNS (gal‧en‧es), which breaks down and recycles cellular waste called glycosaminoglycans (GAGs).

When the body doesn't produce enough of the enzyme, GAGs build up and can potentially cause serious physical problems in

  • Tissues
  • Bones
  • Major organs
Image

Get the details on Morquio A

What is Morquio A?

Morquio A is a form of mucopolysaccharidosis (MPS), a type of lysosomal storage disorder.  Also known as MPS IVA, Morquio A is a rare and progressive inherited disease that affects major organ systems in the body.

What causes Morquio A?

Enzymes are proteins that perform  specific jobs in your body. People with Morquio A do not make enough of a specific enzyme, called N-acetylgalactosamine-6 sulfatase, or GALNS (gal‧en‧es), which breaks down and recycles cellular waste called glycosaminoglycans (GAGs).

Image

When the body doesn't produce enough of the enzyme, GAGs build up and can potentially cause serious physical problems in

  • Tissues
  • Bones
  • Major organs

How do people inherit Morquio A?

Morquio A is a recessively inherited condition. This means that both parents must have a genetic mutation, or variant, in the GALNS gene to pass Morquio A on to their children.

When 2 parents who are carriers have children, each pregnancy presents a 25% chance (1 in 4) of passing Morquio A on to that child.

Recessive inheritance pattern of Morquio A

How do people inherit Morquio A?

Morquio A is a recessively inherited condition. This means that both parents must have a genetic mutation, or variant, in the GALNS gene to pass Morquio A on to their children.

When 2 parents who are carriers have children, each pregnancy presents a 25% chance (1 in 4) of passing Morquio A on to that child.

Since the symptoms of Morquio A may worsen over time, it's important to look for early symptoms to ensure that you get timely treatment to reduce the impact of Morquio A.

Since the symptoms of Morquio A may worsen over time, it's important to look for early symptoms to ensure that you get timely treatment to reduce the impact of Morquio A.

What are the signs and symptoms of Morquio A?

Morquio A is heterogeneous (het·er·oh·gene·ee·ous), which means that it affects each person differently. The signs and symptoms you experience may be different from other people with the condition, and your Morquio A may progress at a different rate.

Individuals with Morquio A may have:

It is important to know that Morquio A does not affect your ability to think and learn.

The symptoms of Morquio A progress over time

Morquio A is progressive, which means that symptoms get worse over time as GAGs build up in the body. 

For many people with Morquio A, symptoms can appear by age 2 or 3, while some show signs and symptoms at 6 months old. Others may not show initial symptoms (including hip stiffness and pain) until the second decade of life.

What are the signs and symptoms of Morquio A?

Morquio A is heterogeneous (het·er·oh·gene·ee·ous), which means that it affects each person differently. The signs and symptoms you experience may be different from other people with the condition, and your Morquio A may progress at a different rate.

Individuals with Morquio A may have:

It is important to know that Morquio A does not affect your ability to think and learn.

The symptoms of Morquio A progress over time

Morquio A is progressive, which means that symptoms get worse over time as GAGs build up in the body. 

For many people with Morquio A, symptoms can appear by age 2 or 3, while some show signs and symptoms at 6 months old. Others may not show initial symptoms (including hip stiffness and pain) until the second decade of life.

    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 BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or go to www.fda.gov/medwatch.

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

    Please see full Prescribing Information, including important warning.