Dany is now receiving Elaprase treatments weekly. Each treatment is a 4 hour long process. This treatment is not a cure, but hopefully will slow down the progression. Clinical trials are ongoing for elaprase treatments that will cross the brain barrier, allowing the elaprase to go directly into the brain. This study is being conducted in North Carolina. The largest issue with this clinical trial, is that the most of the children do not qualify, due to the testing requirements evolved, and the progression of the syndrome has far surpasses the required standards.
For Hunter syndrome. However, enzyme replacement therapy is now available as the deficient enzyme has been produced using recombinant DNA technology, and has been approved for use as a medical drug. The enzyme is administered via intravenous infusion once weekly. This treatment has proven effective for joints and the respiratory tract. It also decreases the size of the liver and improves general health, but as yet it has not been possible to establish any effect on neurological symptoms. International studies are ongoing for follow-up of all treated patients, and for determining the benefits of enzyme replacement therapy in individual organs. It will hopefully be possible to evaluate these studies within the nearest decade. Other interventions aim to prevent complications and ensure the best possible quality of life.
Together Saving Dany
Finding and Funding a Cure for Hunter Syndrome