BioMarin RareConnections™ – BioMarin RareConnections is dedicated to getting you access to VIMIZIM® (elosulfase alfa), and helping you coordinate all the details of your treatment, as well as providing you with other resources to help you manage Morquio A. If you’re interested in this service, give us a call at 1-866-906-6100.
Carrier – A person who has a genetic mutation, but does not show symptoms of a disease. However, they can pass this mutation on to their children.
Cells – Cells are called the “building blocks of life” because they are the substance that makes up all living things. In fact, the human body is made up of 100 trillion cells.
Clinical studies – Scientists use these tests to find out the safety and effectiveness of a medicine.
Endurance – A measure of how long a person can push himself or herself physically. Endurance measures how well your whole body performs—from heart and lungs to bones and muscles.
Enzyme – A protein that performs a specific job in the body.
Enzyme replacement therapy (ERT) – This medical treatment replaces deficient enzymes.
Extension study – A continuation of a study or studies performed by scientists to learn how well a medicine works.
Glycosaminoglycans (GAGs) – Long, sugar carbohydrate chains. Certain GAGs can’t be broken down by the body in someone with Morquio A.
GALNS – People with Morquio A lack the N-acetylgalactosamine-6 sulfatase (GALNS) enzyme, which works to break down GAGs.
Hypersensitivity – Also called a hypersensitivity reaction. This occurs when the body has an extreme response to an allergen or unfamiliar substance. Hypersensitivity reactions range from mild to serious, and they also range in how quickly they develop. Some reactions occur almost immediately, while others may not happen until after exposure to an allergen.
Keratan sulfate (KS) – A type of GAG found in the cornea, bone, and cartilage.
Lysosomal storage disorders – A group of rare, inherited diseases usually involving a malfunctioning enzyme.
Mucopolysaccharide – A kind of sticky molecule, also known as a GAG, that the body uses to build certain tissues. A healthy body continuously breaks down used mucopolysaccharide molecules and recycles them.
Mucopolysaccharidosis (MPS) – People with MPS disorders are not able to break down cellular material in a normal way because they lack the enzymes needed to do so. Morquio A is a kind of MPS disorder. Morquio A is also known as MPS IVA.
Placebo – A placebo is a substance that is made to look like a medication. But, unlike a real medication, placebos don’t contain any active ingredients.
Port – A device that is placed in the chest area or under the skin using local or general anesthetics. The advantage of a port is that it gives doctors and nurses easier access to veins.
Protein –A protein is a molecule that is made up of amino acids. Most enzymes are proteins. They have a lot of important jobs in the body, including helping to make important chemical reactions happen.
Protein-based – Made up of proteins.
Recessive – Traits that are often hidden by dominant traits.
Respiratory – Anything related to breathing.
Six-minute walk test – Also known as the 6MWT, this test measures how far a person can walk in 6 minutes, which is an indicator of endurance.
Tissues – A grouping of the same cell type.
Walking aids – Devices that help people walk, including canes, walkers, and wheelchairs.