New forms of treatment are researched to increase the effectiveness on tissues that do not respond well to the enzyme replacement therapy – such as brain, bones and cartilage.

The aim of the research is to improve the already existing enzyme replacement therapy or to develop new therapeutic approaches. Some of the therapeutic options are still in research, others tested in clinical trials.

Ttreatment of the brain by direct administration into the brain / cerebrospinal fluid

1. Crossing the blood-brain barrier via intrathecal enzyme administration
2. In vivo gene therapy directly into the brain cells

 

Treatment of the brain by direct crossing of the blood-brain barrier

1. Using the transferring receptor mechanism to get past the blood-brain barrier
2. Using the insulin receptors at the blood brain barrier
3. Using nanoparticles to cross the blood-brain barrier

 

Other methods to improve enzyme action or symptom control

1. “Detecting” gene modification in the genetic material responsible for the lacking enzyme production
2. Reducing the inflammatory reaction in joints (pentosane polysulfate PPS)