4 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…
Primary objective: To establish the MSSD of INC424 in patients with MF and baseline PLT count < 100 x 109/L and * 75 x 109/L (first stratum) and PLT count < 75 x 109/L and * 50 x 109/L (second stratum).Secondary objective: safety, PK,…
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…
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.