North Texas teen offers hope to others with Hunter Syndrome
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.
FierceBiotech11d
RegenXBio sells Hunter syndrome gene therapy's U.S., Asia commercial rights for $110M upfront
With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...
University of Manchester3mon
Groundbreaking gene therapy trial for Hunter syndrome opens
“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 ...
Hosted on MSN16d
DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy Designation
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 ...
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 ... emerge and before children ...
Yahoo Finance16d
DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy Designation
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).