RareWays is a research directory. It helps you understand the science. It is not medical advice. Always discuss your care options with your healthcare team.
Coverage: 2015-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
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Mucopolysaccharidosis Type 4: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Exosome-Mediated Intracellular Delivery of N-Acetylgalactosamine-6-Sulfatase and Biochemical Correction in Mucopolysaccharidosis IVA Fibroblasts
Insung Kim et al., Journal of Korean Medical Science (16 October 2026)
https://doi.org/10.3346/jkms.2027.42.e39
- 2.
Haematopoietic stem cell transplant in a case of mucopolysaccharidosis IV with Fanconi anaemia and bone marrow failure.
Mukherjee Swetlana et al., BMJ case reports (11 September 2026)
https://pubmed.ncbi.nlm.nih.gov/42728074/
- 3.
Molecular basis of mucopolysaccharidosis IVA in a large cohort: genotype-phenotype correlations and reporting of 12 novel GALNS variants.
Yin Xi et al., Human molecular genetics (11 September 2026)
https://pubmed.ncbi.nlm.nih.gov/42822866/
- 4.
Two-year follow-up of musculoskeletal outcomes and quality of life in patients with mucopolysaccharidosis type IV and VI.
Genç Emine et al., Journal of pediatric endocrinology & metabolism : JPEM (24 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42333578/
- 5.
Long-Term Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Mucopolysaccharidosis IVA: A 10-Year Follow-Up from a Single-Center Experience in China.
Gong Weiwei et al., Transplantation and cellular therapy (1 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42044726/
Clinical Trials : Australian Sites
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
BMN 110 Phase 3B in Australian Patients
NCT01966029 · Completed · Phase 3 · BioMarin Pharmaceutical
https://clinicaltrials.gov/study/NCT01966029
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 4
Mucopolysaccharidosis type 4, also called Morquio syndrome, is a rare inherited condition in which the body cannot break down certain complex sugars, so they build up in bone and cartilage. It mainly affects the skeleton and joints, and can also involve the spine, heart, hearing, eyes and breathing. Care involves a specialist team, with enzyme replacement therapy available for the type A form.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
There is not much recent research on Mucopolysaccharidosis Type 4, so this page includes research published since 2015. Most pages on RareWays start at 2020.
Most Recent Research
Common Questions
What is Mucopolysaccharidosis Type 4?
Mucopolysaccharidosis type 4, also called Morquio syndrome, is a rare inherited condition in which the body cannot break down certain complex sugars, so they build up in bone and cartilage. It mainly affects the skeleton and joints, and can also involve the spine, heart, hearing, eyes and breathing. Care involves a specialist team, with enzyme replacement therapy available for the type A form.
How many clinical trials are available for Mucopolysaccharidosis Type 4?
RareWays currently indexes 12 clinical trials for Mucopolysaccharidosis Type 4, of which 1 is actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Mucopolysaccharidosis Type 4 come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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