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The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
Harmatz P, Hendriksz CJ, Lampe C, McGill JJ, Parini R, Leão-Teles E, Valayannopoulos V, Cole TJ, Matousek R, Graham S, Guffon N, Quartel A; MPS VI Study Group. Harmatz P, et al. Mol Genet Metab. 2017 Sep;122(1-2):107-112. doi: 10.1016/j.ymgme.2017.03.008. Epub 2017 Mar 31. Mol Genet Metab. 2017. PMID: 28457718 Free article. Clinical Trial.
Spinal cord issues in adult patients with MPS: transition of care survey.
Ghotme KA, Alvarado-Gomez F, Lampe C, White KK, Solano-Villareal M, Giugliani R, Harmatz PR. Ghotme KA, et al. Among authors: harmatz pr. Childs Nerv Syst. 2018 Sep;34(9):1759-1765. doi: 10.1007/s00381-018-3834-6. Epub 2018 May 27. Childs Nerv Syst. 2018. PMID: 29804213
Spinal involvement in mucopolysaccharidosis IVA (Morquio-Brailsford or Morquio A syndrome): presentation, diagnosis and management.
Solanki GA, Martin KW, Theroux MC, Lampe C, White KK, Shediac R, Lampe CG, Beck M, Mackenzie WG, Hendriksz CJ, Harmatz PR. Solanki GA, et al. Among authors: harmatz pr. J Inherit Metab Dis. 2013 Mar;36(2):339-55. doi: 10.1007/s10545-013-9586-2. Epub 2013 Feb 6. J Inherit Metab Dis. 2013. PMID: 23385297 Free PMC article. Review.
Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure.
Langereis EJ, Borgo A, Crushell E, Harmatz PR, van Hasselt PM, Jones SA, Kelly PM, Lampe C, van der Lee JH, Odent T, Sakkers R, Scarpa M, Schafroth MU, Struijs PA, Valayannopoulos V, White KK, Wijburg FA. Langereis EJ, et al. Among authors: harmatz pr. Orphanet J Rare Dis. 2013 Oct 3;8:155. doi: 10.1186/1750-1172-8-155. Orphanet J Rare Dis. 2013. PMID: 24088413 Free PMC article. Review.
Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I.
Kovac V, Shapiro EG, Rudser KD, Mueller BA, Eisengart JB, Delaney KA, Ahmed A, King KE, Yund BD, Cowan MJ, Raiman J, Mamak EG, Harmatz PR, Shankar SP, Ali N, Cagle SR, Wozniak JR, Lim KO, Orchard PJ, Whitley CB, Nestrasil I. Kovac V, et al. Among authors: harmatz pr. Mol Genet Metab. 2022 Feb;135(2):122-132. doi: 10.1016/j.ymgme.2022.01.001. Epub 2022 Jan 7. Mol Genet Metab. 2022. PMID: 35012890 Free PMC article.
82 results