In some cases of MPS I and MPS VI, there may not be clinically clouding of corneae, and can have features overlapping with MPS II or Hunter syndrome. Manifestations can be subtle and diagnosis ...

Hunter syndrome results from the absence of an enzyme needed to break down cellular waste. Without it, waste builds up in the body, causing progressive damage to various systems.

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...

“Mice with Hunter syndrome treated with the HSC gene therapy showed dramatic improvement in their condition, including normalization of working memory problems, and skeletal features such as the ...

Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter ...

dDSA confirmed the CE-MRA findings, leading to the diagnosis of Bow Hunter’s syndrome (BHS) with bilateral dynamic stenosis of VAs (figures 2 and 3). The examination also revealed the presence of a ...

The University of Manchester has announced today a groundbreaking gene therapy partnership to ease the lifelong suffering of people with Hunter syndrome. The University ... emerge and before children ...

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).