2 results
Approved WMOCompleted
The study has three objectives. Firstly, we will study the safety and tolerability of the drug after the administration of both single and multiple doses of the drug. Secondly, we will study the speed at which the drug is absorbed in the body, as…
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…