Denali is conducting the following Hunter syndrome clinical trials
A Study of DNL310 in Pediatric Subjects with Hunter syndrome
A Phase 1/2 study of DNL310 for pediatric patients with Hunter syndrome (MPS II). The study includes a 24-week dose escalation phase with an 18-month safety extension and will be conducted at multiple sites.
Study Status: This study is currently recruiting participants. For additional information, please view details on Clinicaltrials.gov
Policy On Expanded Access To Investigational Drugs
Denali understands that there are some cases when a patient is unable to participate in a clinical trial, and other treatment options have been exhausted. In those cases the patient’s physician may choose to request access to an investigational drug outside of a clinical trial via what is termed Expanded Access in the United States.
Currently, participation in clinical trials is the only way for patients to gain access to Denali’s investigational therapies. As more clinical data on the safety and efficacy of these investigational therapies become available, we will review and update our policy on Expanded Access.