3 results
Primary ObjectiveThe primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with late-onset Pompe disease.Secondary ObjectivesThe secondary…
To compare one dose of the short acting tropicamide combined with one dose of the longer acting cyclopentolate (c+t) with a double dose of the longer acting cyclopentolate (c+c). To develop a cycloplegics protocol that garantees optimal refractieve…
The primary objective of this study is to provide extended access to momelotinib (MMB) and assess long-term safety in 4 cohorts of subjects who are currently receiving treatment with MMB and have not experienced progression of disease:* Cohort 1:…