2 results
Approved WMORecruiting
• To assess feasibility and safety of a sequential treatment regime in which standard intensive chemotherapy (fludarabin-amsacrin-cytarabin) is directly followed by standard allogeneic stem cell transplantation (T cell depleted RIC alloSCT with…
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…