with Mucopolysaccharidosis Type II (MPS II), which is also known as Hunter syndrome. The pivotal phase of the study met its primary endpoint of patients achieving a reduction in cerebrospinal ...

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).

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

About DNL310 and Hunter Syndrome (MPS II) Hunter syndrome (MPS II) is a rare neurodegenerative lysosomal storage disease caused by mutations in the gene that encodes for the enzyme iduronate-2 ...

About Hunter Syndrome (MPS II) Hunter syndrome (MPS II) is a rare genetic disease that affects over 2,000 individuals in commercially accessible geographies, primarily males, and leads to physical ...

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome ... one year of age with the condition also known as ...

Shire’s attempts to develop a drug for cognitive impairment from the rare inherited disease Hunter Syndrome has failed ... and families living with MPS II.” “We are grateful to the children ...

With a regulatory decision expected before the end of this year, RGX-121 could become the first gene therapy approved for Hunter syndrome. Also known as mucopolysaccharidosis type II, Hunter ...

for the treatment of individuals with Hunter syndrome (MPS II). This designation is in addition to Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation ...