We plan for this study to assist in the design of clinical research trials to help test potential treatments
for Mucolipidosis Type IV. Clinical research trials are trials of treatments in people with the disease.
Before clinical trials are approved by the FDA, we must present a highly developed picture of the disease
progression at all stages and ages to the FDA, indicating to them that we understand Mucolipidosis Type
IV fully. This demonstrates that we understand how and when a treatment will be effective. For this
reason, your participation in research studies like this are crucial to the development of effective
JOIN US TO HELP PHYSICIANS GATHER CLINICAL PATIENT DATA FOR MUCOLIPIDOSIS TYPE IV. THIS
DATA IS ESSENTIAL IN ORDER TO BUILD MEANINGFUL TREATMENTS!