3 results
Primary Objective: To determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary Objectives: Safety, formula acceptability and…
The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704.
As patients with congenital vascular malformations often suffer from severe pain and morbidity due to the vascular malformation, quality of life is often signifcantly impaired. The primairy objective of the present study is to reduce pain complaints…