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 ...

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 found a commercialization partner for both the U.S. and Asian markets.

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

Hunter syndrome can be diagnosed using the physical symptoms of children at the age of 18 months and 4 years. Treatments can help manage condition, no known cure Diagnosed by medical professional ...

GC Green Cross announced on the 11th that its severe Hunter syndrome treatment ‘Hunterase ICV’ has received product approval from the Russian Federal Ministry of Health. This is the first ...

We are grateful to the FDA for recognizing the potential of tividenofusp alfa as a meaningful treatment option for individuals with Hunter syndrome. We look forward to continued collaboration with ...