pharmaphorum4d
Regenxbio expands MPS I gene therapy trial after first look at data
Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...
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Denali Therapeutics to Present Interim Data from Phase 1/2 Study of ETV:IDS (DNL310) for the Potential Treatment of Hunter Syndrome at MPS 2021
Details related to Denali’s oral presentation at MPS 2021 and a webinar ... More information about the ongoing Phase 1/2 study of DNL310 in patients with Hunter syndrome can be found on ...