TürkiyeResearch and clinical trials
MPS IVA and VI patients on enzyme therapy in Istanbul: leg findings improved slightly over two years
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In Istanbul, 14 MPS patients receiving enzyme therapy were followed for two years. Six had Morquio A (MPS IVA) and eight had MPS VI. The musculoskeletal finding score for the legs decreased significantly. There was no change in arm and spine findings. Quality of life scores did not change either.
What does it mean for patients and families?
Regular enzyme therapy may partly keep some musculoskeletal findings stable. However, no marked improvement in quality of life was seen. The study is small.
Source
Scientific paper: Two-year follow-up of musculoskeletal outcomes and quality of life in patients with mucopolysaccharidosis type IV and VI. Journal of Pediatric Endocrinology & Metabolism, 2026. DOI: 10.1515/jpem-2025-0665 · PubMed 42333578
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Morquio A syndrome (MPS IVA)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.