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

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

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.

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 ...

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 ...

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 ...

Shire’s attempts to develop a drug for cognitive impairment from the rare inherited disease Hunter Syndrome has failed to produce results. The company’s Elaprase (idursulfase) is already ...

Bow Hunter's Syndrome (BHS) is a rare condition characterized by vertebrobasilar insufficiency, which occurs when the vertebral artery is compressed during head rotation. This compression can lead ...

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 ...