The Denali Approach

Brain delivery is a critical unmet need in Hunter syndrome therapy1

Treatments that cross the blood-brain barrier and aim to treat the neurocognitive components of Hunter syndrome, such as developmental delay, disruptive behavior and impaired cognition, remain a critical unmet medical need.

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References: 1. R. J. Desnick, E. H. Schuchman, Enzyme replacement therapy for lysosomal diseases: Lessons from 20 years of experience and remaining challenges. Annu. Rev. Genomics Hum. Genet. 13, 307–335 (2012). 2. Kariolis et al., Sci. Transl. Med. 12, eaay1359 (2020) 27 May 2020 3. Ullman et al., Sci. Transl. Med. 12, eaay1163 (2020) 27 May 2020

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