{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T14:46:52Z","timestamp":1786978012306,"version":"build-2736575974"},"reference-count":30,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2024,3,11]],"date-time":"2024-03-11T00:00:00Z","timestamp":1710115200000},"content-version":"vor","delay-in-days":10,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2024,3]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Lysosomal enzyme deficiency in mucopolysaccharidosis (MPS) I results in glycosaminoglycan (GAG) accumulation leading to pain and limited physical function. Disease\u2010modifying treatments for MPS I, enzyme replacement, and hematopoietic stem cell therapy (HSCT), do not completely resolve MPS I symptoms, particularly skeletal manifestations. The GAG reduction, anti\u2010inflammatory, analgesic, and tissue remodeling properties of pentosan polysulfate sodium (PPS) may provide disease\u2010modifying treatment for musculoskeletal symptoms and joint inflammation in MPS I following ERT and\/or HSCT. The safety and efficacy of PPS were evaluated in four subjects with MPS I aged 14\u201319\u2009years, previously treated with ERT and\/or HSCT. Subjects received doses of 0.75\u2009mg\/kg or 1.5\u2009mg\/kg PPS via subcutaneous injections weekly for 12\u2009weeks, then every 2\u2009weeks for up to 72\u2009weeks. PPS was well tolerated at both doses with no serious adverse events. MPS I GAG fragment (UA\u2010HNAc [1S]) levels decreased at 73\u2009weeks. Cartilage degradation biomarkers serum C\u2010telopeptide of crosslinked collagen (CTX) type I (CTX\u2010I) and type II (CTX\u2010II) and urine CTX\u2010II decreased in all subjects through 73\u2009weeks. PROMIS scores for pain interference, pain behavior, and fatigue decreased in all subjects through 73\u2009weeks. Physical function, measured by walking distance and dominant hand function, improved at 49 and 73\u2009weeks. Decreased GAG fragments and cartilage degradation biomarkers, and positive PROMIS outcomes support continued study of PPS as a potential disease\u2010modifying treatment for MPS I with improved pain and function outcomes.<\/jats:p>","DOI":"10.1002\/jimd.12715","type":"journal-article","created":{"date-parts":[[2024,3,11]],"date-time":"2024-03-11T23:53:50Z","timestamp":1710201230000},"page":"355-365","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Open\u2010label, single\u2010center, clinical study evaluating the safety, tolerability and clinical effects of pentosan polysulfate sodium in subjects with mucopolysaccharidosis I"],"prefix":"10.1002","volume":"47","author":[{"given":"Drago","family":"Bratkovic","sequence":"first","affiliation":[{"name":"Metabolic Unit Women's and Children's Hospital  North Adelaide South Australia Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Curtis","family":"Gravance","sequence":"additional","affiliation":[{"name":"Paradigm Biopharmaceuticals Ltd.  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