pharmaphorum7y
Shire's Hunter Syndrome cognitive impairment drug fails
Shire’s attempts to develop a drug for cognitive impairment from the rare inherited disease Hunter Syndrome has failed ... senior vice president and global head of R&D (ad-interim), Shire ...
WGN Radio1mon
Denali Therapeutics Announces Primary Analysis and Long-Term Follow-Up of Phase 1/2 Study in Hunter Syndrome (MPS II) with Tividenofusp Alfa
Feb. 06, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced the primary analysis of the Phase 1/2 study in 47 participants with Hunter syndrome (MPS II) in the 24-week ...
University of Manchester4y
University announces partnership with AVROBIO for Hunter syndrome gene therapy
The University of Manchester has announced today a groundbreaking gene therapy partnership to ease the lifelong suffering of people with Hunter syndrome. The University ... and facilitate uptake by ...
University of Manchester5mon
Groundbreaking gene therapy trial for Hunter syndrome opens
The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare ... The modified HSCs are then ...