NEJM Publishes Phase 1/2 Results for Denali Therapeutics’ Brain‑Penetrant Hunter Syndrome Therapy

A newly published Phase 1/2 study in The New England Journal of Medicine, and reported on by the Manila Times, highlights promising early results for tividenofusp alfa (DNL310), Denali Therapeutics’ investigational…

Continue Reading NEJM Publishes Phase 1/2 Results for Denali Therapeutics’ Brain‑Penetrant Hunter Syndrome Therapy
FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome
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FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome

According to an article published in Yahoo, the FDA has recently granted the Rare Pediatric Disease designation to AVR-RD-05, a gene therapy for Hunter syndrome. This designation is reserved for…

Continue Reading FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome