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.
FierceBiotech12d
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 ...
Hosted on MSN17d
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 Manchester3mon
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 lysosomal storage disorder. Five ...
Yahoo Finance17d
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 ...