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Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.
Dickson PI, Kaitila I, Harmatz P, Mlikotic A, Chen AH, Victoroff A, Passage MB, Madden J, Le SQ, Naylor DE; Mucopolysaccharidosis I Intrathecal Research Collaborative. Dickson PI, et al. Mol Genet Metab. 2015 Sep-Oct;116(1-2):69-74. doi: 10.1016/j.ymgme.2015.07.005. Epub 2015 Jul 26. Mol Genet Metab. 2015. PMID: 26260077 Free PMC article. Clinical Trial.