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Page 1
Recommendations for the use of sapropterin in phenylketonuria.
Cunningham A, Bausell H, Brown M, Chapman M, DeFouw K, Ernst S, McClure J, McCune H, O'Steen D, Pender A, Skrabal J, Wessel A, Jurecki E, Shediac R, Prasad S, Gillis J, Cederbaum S. Cunningham A, et al. Among authors: jurecki e. Mol Genet Metab. 2012 Jul;106(3):269-76. doi: 10.1016/j.ymgme.2012.04.004. Epub 2012 Apr 13. Mol Genet Metab. 2012. PMID: 22575621 Free article. Clinical Trial.
Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome.
Hendriksz CJ, Parini R, AlSayed MD, Raiman J, Giugliani R, Solano Villarreal ML, Mitchell JJ, Burton BK, Guelbert N, Stewart F, Hughes DA, Berger KI, Slasor P, Matousek R, Jurecki E, Shaywitz AJ, Harmatz PR. Hendriksz CJ, et al. Among authors: jurecki e. Mol Genet Metab. 2016 Sep;119(1-2):131-43. doi: 10.1016/j.ymgme.2016.05.018. Epub 2016 Jun 16. Mol Genet Metab. 2016. PMID: 27380995 Free article. Clinical Trial.
Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome.
Hendriksz CJ, Berger KI, Parini R, AlSayed MD, Raiman J, Giugliani R, Mitchell JJ, Burton BK, Guelbert N, Stewart F, Hughes DA, Matousek R, Jurecki E, Decker C, Harmatz PR. Hendriksz CJ, et al. Among authors: jurecki e. J Inherit Metab Dis. 2016 Nov;39(6):839-847. doi: 10.1007/s10545-016-9973-6. Epub 2016 Aug 23. J Inherit Metab Dis. 2016. PMID: 27553181 Free PMC article. Clinical Trial.
28 results