3 results
Approved WMOCompleted
To assess PK of MPhs and MP.
Approved WMOCompleted
To evaluate the efficacy, safety, tolerability, in subjects with ALS following the oral administration of study drug (active or placebo capsules).
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…