2 results
Approved WMOCompleted
Parts D and E: Primary Objectives:• To evaluate the safety and tolerability of VX-445 in TC with TEZ and IVA in subjects with cystic fibrosis (CF)• To evaluate the efficacy of VX-445 in TC with TEZ and IVA in subjects with CFSecondary Objectives• To…
Approved WMORecruiting
The primary objectives are: To evaluate the CNS activity of DNL310 vs idursulfase as measured by the cerebrospinal fluid (CSF) concentration of heparan sulfate (HS) in participants with the neuronopathic form of mucopolysaccharidosis type II (nMPS…