Denali ERT clinical trial, Sanfilippo Type A, UCSF site activated

December 7, 2023

Cure Sanfilippo Foundation is happy to share that we’ve been notified by the institution that a clinical trial site for Sanfilippo Type A has opened at the University of California-San Francisco (UCSF) in the U.S.

This trial is sponsored by Denali Therapeutics. It will deliver enzyme into the bloodstream and is specifically formulated to be able to cross from the blood-brain barrier.

Below are some of the key eligibility criteria for this first two cohorts of the study. Additional eligibility criteria and information will become available soon on the website.

Key Inclusion Criteria include, but are not limited to the following:

  • Cohort A: Patients ≥ 2 to < 18 years old at screening with a documented diagnosis of MPS IIIA (BEGINNING ENROLLEMENT)
  • Cohort B: Patients > 6months and <2 years at screening with a severe phenotype (NOT YET ENROLLING)
  • Key Exclusion Criteria include, but are not limited to, the following:
    • Patients who have lost the ability to walk independently
    • Patients who are unable to take the majority of nutrition via mouth
    • Patients who have history of gene therapy or HSCT (stem cell transplant)

To learn more or express your interest in participating in the clinical trial, you may contact: Ella Rushing (study coordinator) 510-428-3885, ext. 4785, or

Information about Denali’s enzyme replacement program was presented this August at Cure Sanfilippo Foundation’s ADVANCE 2023 conference and can be viewed below.

Additionally, we at Cure Sanfilippo Foundation are here to provide support as you consider your options and to assist you with information and getting you connected. Please feel free to reach out to us (

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