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

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

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.

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 ...

One of the treatments, for a progressive disorder known as Hunter syndrome, could secure approval in the U.S. as soon as late ...

An X-linked genetic disease caused by the deficiency ... difficulty talking and ear infections are the commonly noted symptoms. Hunter syndrome can be diagnosed using the physical symptoms of ...

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, which affects an estimated one in 100,000 males ... The University of Manchester Foundation Trust to develop a therapy for this debilitating genetic disease. AVROBIO’s strategic focus ...

In a deal potentially worth $810 million for Regenxbio Inc., Nippon Shinyaku Co. Ltd. is partnering on the U.S. and Asian development and commercialization of iduronate-2-sulfatase enzyme RGX-121 for ...

In 2021, the FDA granted Fast Track designation to tividenofusp alfa for the treatment of patients with Hunter syndrome ... mutations confirmed by genetic testing. BIIB122 is also being evaluated ...