6533b7dcfe1ef96bd1272038

RESEARCH PRODUCT

Safety study of sodium pentosan polysulfate for adult patients with mucopolysaccharidosis type II

Toshiyuki FukaoMolly StapletonShunji TomatsuShunji TomatsuKenji E. OriiEdward H. SchuchmanYasuyuki SuzukiTadashi MatsumotoCalogera M. SimonaroAlaena Lim

subject

Drug0301 basic medicinemedicine.medical_specialtymedia_common.quotation_subjectEndocrinology Diabetes and MetabolismClinical BiochemistryeducationUrologymucopolysaccharidosis IIBiochemistryArticlePPSrange of motion03 medical and health sciences0302 clinical medicineEndocrinologyInternal medicineClinical endpointglycosaminoglycanGeneticsMedicineMucopolysaccharidosis type IIAdverse effectMolecular Biologyhealth care economics and organizationsmedia_commonbiologyAdult patientsanti-inflammatory factorbusiness.industryEnzyme replacement therapyPentosan polysulfateClinical trial030104 developmental biologyAlanine transaminasebiology.proteinSodium Pentosan Polysulfatebusiness030217 neurology & neurosurgerymedicine.drug

description

Current therapies for the mucopolysaccharidoses (MPS) do not effectively address skeletal and neurological manifestations. Pentosan polysulfate (PPS) is an alternative treatment strategy that has been shown to improve bone architecture, mobility, and neuroinflammation in MPS animals. The aims of this study were to a) primarily establish the safety of weekly PPS injections in attenuated MPS II, b) assess the efficacy of treatment on MPS pathology, and c) define appropriate clinical endpoints and biomarkers for future clinical trials. Subcutaneous injections were administered to three male Japanese patients for 12 weeks. Enzyme replacement therapy was continued in two of the patients while they received PPS and halted for two months in one patient before starting PPS. During treatment, one patient experienced an elevation of alanine transaminase, and another patient experienced convulsions

yearjournalcountryeditionlanguage
2019-12-17Molecular Genetics and Metabolism
https://doi.org/10.1016/j.ymgme.2015.12.384