After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Created with Sketch. This rare sex chromosome abnormality, also referred to as Jacob’s syndrome, occurs when a male infant is born with an extra Y chromosome. Some symptoms include being tall ...

Despite having gone through this before with her first son, Yusof, and despite the doctors explaining that this was a gene ...

Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued ...

“We expect the progress achieved in our Hunter syndrome program to inform and ... M.D., Professor of Pediatrics, Genetics, Genomics and Metabolism at Feinberg School of Medicine in Chicago ...

and women born with just one X chromosome (a diagnosis known as Turner Syndrome), can have cognitive impairments. X chromosomes, some researchers hypothesize, could help explain sex differences in ...

Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).

A Denali Therapeutics drug for the rare enzyme deficiency Hunter syndrome is still in pivotal testing, but the company has guidance from the FDA on a pathway to get this therapy to the market sooner.

Denali also supported additional research relating to Hunter syndrome (MPS II) at the WORLDSymposium™ conference. Research presented by Barbara Burton, M.D., Professor of Pediatrics ...

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 ...