Mucopolysaccharidosis Type II — Research Summary
Printed from RareWays (rareways.com.au) on 26 July 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Dynamic Angiography Demonstrating Discogenic Bow Hunter Syndrome.
Sharashidze Vera et al. — Stroke (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42330130/
- 2.
Dynamic bilateral vertebral artery occlusion owing to os odontoideum mimicking basilar artery occlusion: illustrative case.
Morita Takao et al. — Journal of neurosurgery. Case lessons (29 June 2026)
https://pubmed.ncbi.nlm.nih.gov/42372306/
- 3.
Tividenofusp Alfa: First Approval.
Shirley Matt — Molecular diagnosis & therapy (18 June 2026)
https://pubmed.ncbi.nlm.nih.gov/42313339/
- 4.
Impact of Mucopolysaccharidosis Type II in Young Children from the Caregiver's Perspective: A Qualitative Study.
Baldwin Jessica et al. — Advances in therapy (12 June 2026)
https://pubmed.ncbi.nlm.nih.gov/42283955/
- 5.
Development of Dried Blood Spot Proficiency Testing Materials for Newborn Screening of Lysosomal Diseases Using Recombinant Enzymes.
Courtney Elya et al. — International journal of neonatal screening (9 June 2026)
https://pubmed.ncbi.nlm.nih.gov/42346729/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Recruiting — Phase 2 — Denali Therapeutics Inc.
https://clinicaltrials.gov/study/NCT05371613
- 2.
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Recruiting — Phase 2 — Ultragenyx Pharmaceutical Inc
https://clinicaltrials.gov/study/NCT02716246
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Mucopolysaccharidosis Type II
Mucopolysaccharidosis type II (Hunter syndrome) is an X-linked lysosomal storage disorder caused by IDS enzyme deficiency. It predominantly affects males and causes progressive multi-organ involvement including cognitive decline, joint stiffness, and cardiac disease. Enzyme replacement therapy is established, and gene therapy trials are ongoing.
Most Recent Research
Common Questions
What is Mucopolysaccharidosis Type II?
Mucopolysaccharidosis type II (Hunter syndrome) is an X-linked lysosomal storage disorder caused by IDS enzyme deficiency. It predominantly affects males and causes progressive multi-organ involvement including cognitive decline, joint stiffness, and cardiac disease. Enzyme replacement therapy is established, and gene therapy trials are ongoing.
How many clinical trials are available for Mucopolysaccharidosis Type II?
RareWays currently indexes 61 clinical trials for Mucopolysaccharidosis Type II, of which 6 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Mucopolysaccharidosis Type II come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is updated regularly by Rocky, RareWays' automated research engine. All articles and trials link directly to their original sources.
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This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.