Tag Archives: mucopolysaccharidosis type II

Breaking Barriers Hunter Syndrome Trial

 

 

Researchers are conducting a Hunter Syndrome trial of AGT-182, an investigational treatment for people with mucopolysaccharidosis type 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.

 

To sign up for the Breaking Barriers Hunter Syndrome trial, sign up here.

What is Hunter Syndrome?

What is Hunter Syndrome?

  • Hunter syndrome, also known as mucopolysaccharidosis type II, or MPS 2, is a rare, hereditary metabolic disorder that primarily affects males; it is often severe and almost always progressive.
  • In people with MPS syndrome, the  iduronate-2-sulfatase (IDS) enzyme, which  breaks down complex sugars, is missing or not working properly.
  • This leads to an abnormal buildup of complex sugars in tissues throughout the body, including the brain, skeleton, joints, spinal cord, heart spleen, or liver.
  • Physical symptoms include growth delay, joint stiffness and coarsening of facial features
  • When severe, MPS disease can cause respiratory and cardiac problems, enlargement of the liver and spleen, and premature death.
  • Hunter syndrome can also cause neurological deficits that can lead to progressive dementia and a progressive decrease in IQ points.