Tag Archives: mucopolysaccharidosis ii

What Is Hunter Syndrome?

 

What is Hunter Syndrome? Hunter Syndrome (also known as mucopolysaccharidosis II or MPS II) is a disorder that occurs when an enzyme the body needs is either missing or it does not work properly. If the body does not have enough enzymes, it cannot break down the kind of sugar that builds bones, skin, tendon, and other tissue. Hunter syndrome eventually causes permanent, progressive damage affecting the appearance, mental development, organ function, and physical abilities of a child.

 

This rare genetic disorder affects mostly males as young as 18 months and has no known cure. The current treatment for Hunter Syndrome involves the management of symptoms, but it has many complications.

What are the symptoms of Hunter Syndrome?

The symptoms of Hunter Syndrome vary person to person. It’s not always present at birth, but indications of this syndrome are known to show up between 2 and 4 years of age. If the symptoms occur at an early age it is usually more severe.

what is Hunter Syndrome InfoGraphic

What causes MPS II?

Children who suffer from Hunter Syndrome inherit the disease from their parents. When a chromosome is missing or malfunctioning, it is usually because the mother was carrying MPS II. However, a father suffering from Hunter Syndrome can also pass it to his daughter who will eventually pass it to her son.

 

 

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RECENT STUDY UPDATE! Click here to download the Newsletter:
Study Update from Armagen 3-18-16

Researchers are conducting a clinical trial of AGT-182, an investigational treatment for people with Hunter Syndrome aka mucopolysaccharidosis II or MPS 2.

AGT-182 is an enzyme replacement therapy (ERT) designed to treat both the body-related (somatic) and central nervous system (CNS) symptoms and complications of Hunter syndrome.

Currently approved treatments for Hunter syndrome (or MPS 2) are unable to treat the brain (CNS) because of the enzyme’s inability to cross the blood-brain barrier (BBB). The BBB is a filter that protects the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. As a result, available therapies for MPS disease do not address many of the severe and progressive neurological complications of the Hunter Syndrome. AGT-182 is designed to cross the BBB in the same way insulin does.

Please CLICK HERE to find out how you or a loved one can participate in this trial.

Safety Information
This Hunter Syndrome trial is monitored and approved by a Data Monitoring Committee (DMC) and safety will be reported to an institutional review board to ensure the safety of the participants.