3 results
Approved WMOCompleted
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…
Approved WMOCompleted
Primary: Evaluate the efficacy of patient-derived tumor organoids to successfully allocate patients for treatment with specific targeted agents.Secondary: Characterize safety and tolerability per experimental treatment regimen.
Approved WMOCompleted
To evaluate in a feasibility study whether low uptake on FES-PET at baseline is related to non-response to letrozole plus palbociclib treatment.