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Research PubMed / J Pediatr Endocrinol Metab clinical-researchpediatric-ptoutcome-measures

Two-year follow-up of musculoskeletal outcomes and quality of life in patients with mucopolysaccharidosis type IV and VI.

PubMed / J Pediatr Endocrinol Metab · 2026-06-24 · By Genç E, Bilekyiğit Kurt C, Evrim Karadağ Saygi N, et al. · Journal of pediatric endocrinology & metabolism : JPEM

A two-year follow-up study examined musculoskeletal function and quality of life in pediatric patients diagnosed with mucopolysaccharidosis (MPS) types IV and VI, rare lysosomal storage disorders that cause progressive joint and skeletal deterioration. Researchers tracked physical outcomes over time, providing data relevant to rehabilitation professionals managing these complex pediatric cases. The findings offer insight into functional trajectories that may inform PT assessment and intervention planning for this population.

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