Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...

One notable study investigated the use of intrathecal idursulfase-IT, an enzyme replacement therapy ... for patients with neuronopathic MPS II (Hunter syndrome). Although the primary endpoint ...

The University of Manchester will act as trial sponsor. Children with Hunter syndrome have a missing gene, meaning they cannot produce an important enzyme called iduronate-2-sulfatase or IDS. The gene ...

SHP609 was supposed to correct the deficiency in the iduronate-2-sulfatase enzyme in the CNS and prevent the cognitive decline seen in some Hunter Syndrome cases. Sadly, Shire said the phase 2/3 ...

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).

An X-linked genetic disease caused by the deficiency of enzyme iduronate-2-sulfatase ... infections are the commonly noted symptoms. Hunter syndrome can be diagnosed using the physical symptoms ...

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. Get top local ...

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.