HealthPrep on MSN11mon
How To Treat Hunter Syndrome
Hunter syndrome is an exceedingly rare genetic disorder caused by a malfunctioning or missing enzyme. Patients with Hunter ...
pharmaphorum7y
Shire's Hunter Syndrome cognitive impairment drug fails
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 ...
University of Manchester5mon
Groundbreaking gene therapy trial for Hunter syndrome opens
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 ...
Hosted on MSN2mon
DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy Designation
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).
조선일보1mon
GC Biopharma designates Hunter syndrome treatment as orphan drug in South Korea
Hunter syndrome is a congenital rare disease caused by a deficiency of the IDS (Iduronate-2-sulfatase) enzyme, leading to skeletal abnormalities and intellectual disability. It is reported to ...
WGN Radio1mon
Denali Therapeutics Announces Primary Analysis and Long-Term Follow-Up of Phase 1/2 Study in Hunter Syndrome (MPS II) with Tividenofusp Alfa
Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued improvement in hearing, cognition and adaptive behavior ...