Tag Archives: Breaking Barriers Hunter Syndrome Trial

Hunter Syndrome Treatments

 

Hunter Syndrome Treatments

Catching Hunter Syndrome at an early stage is crucial for treatment. A child can undergo a bone marrow transplant where bone marrow is taken from the hip of a donor and transplanted into a child. This treatment may help lessen breathing difficulties or problems with mobility, as well as improve heart, liver, and spleen functions. It can also prevent a child’s mental regression.

 

Another possible treatment option is enzyme therapy. This therapy replaces the missing or defective enzymes of a child with man-made or genetically engineered enzymes, which eases many symptoms of Hunter Syndrome. Unfortunately, this treatment comes with some risks and has side effects to the body including headache, fever, skin reactions, and high blood pressure.

 

A third option is gene therapy, which effectively replaces the chromosome for producing the missing enzyme of the body.

 

What are the complications of Hunter Syndrome treatments?

There are several things to consider when choosing the right therapy for a child as each comes with considerable complications. For example, relief for respiratory complications the tonsils and adenoids are removed, but as the disease progresses the tissues thicken and respiratory complications could return. Addressing heart complications could result in surgery to replace heart valves, which comes with its own set of dangers to be aware of.

 

Other complications arise when treating connective tissue problems, managing behavioral problems, and addressing sleep difficulties. Physical therapy will improve joint flexibility, but it will not stop the progression of joint motion. Behavioral problems or issues with sleep can be managed through medication, but these medications could in fact lead to other complications with the disease.

 

How can we manage Hunter Syndrome?

Talk to your doctor, first. There are genetic tests that are available if you believe you could be a carrier of Hunter Syndrome. Second, be positive! A child suffering from Hunter Syndrome needs encouragement and love. Be sure to keep the child active so that the joints remain flexible and mobile. And finally, educate and learn. Nothing is more satisfying to a child than learning, so teach them as much as you can while the brain is still functioning properly. Learn for yourself, as well. There are plenty of online communities available to you and your family to help educate you on Hunter Syndrome.

This is only a brief overview of currently-approved and available hunter syndrome treatments. Info from Breaking Barriers Hunter Syndrome Trial.

 

Hunter Syndrome Trial Update 3-18-2016

 

The Breaking Barriers Hunter Syndrome Trial has successfully completed enrollment for the first cohort/group of four patients! We are now enrolling for the second cohort. Armagen plans to enroll 12 patients that are 18 years and older into this study. If you would like to view the full study update, you can download it below:
Study Update from Armagen 3-18-16

 

Armagen is developing therapies to non-invasively deliver drugs across the blood-brain barrier, a filter that protect the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. Available medications for Hunter Syndrome/MPS II do not cross the blood-brain barrier in clinically relevant amounts and therefore do not address the progress neurological complications of the disease.

 

If you would like to enroll in the Breaking Barriers Hunter Syndrome Trial, you may sign up here.

Hunter Syndrome Clinical Trial Underway!

 

The Breaking Barriers Hunter Syndrome Trial is taking place at Emory University in Atlanta, GA and at many other locations world-wide. Take a look at the research behind this clinical study of Hunter Syndrome.

 

If you would like to sign up for the Breaking Barriers Hunter Syndrome trial, you can do so here.

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.