Current Treatment Options

The current treatment is an Enzyme Replacement Therapy (ERT). It is administered during weekly infusion treatments through a surgically placed port near the heart. Treatments typically last 6 hours, and the intent is to provide the body with the enzyme needed (which we produce normally, but Robert does not) in order for Robert to break down the sugars that accumulate and cause the various health affects to his heart, lungs, eyes, trachea, and the bones.

The current treatment, Enzyme Replacement Therapy (ERT), is inadequate for the following reasons

1. By and large has no meaningful impacts on many of the severe aspects of the disease (including the ongoing damage to the bones, which prevents Robert from growing)
2. Positive effects on energy levels and some soft tissue issues but those positive impacts can be limited for multiple reasons
3. Extremely inconvenient for a child to give up most of a day every week, let alone having to sit in a hospital or at home for 6-7 hours while other children are at school
4. Incredibly expensive
5. Can elicit unintended immune responses
6. Might be completely out of a Morquio patient’s system in about 24 hours (at which point the body will not have anything to assist it until a new treatment can be administered, a week later), as is suggested by some studies.