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3,994 results

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Page 1
Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study.
Hendriksz CJ, Burton B, Fleming TR, Harmatz P, Hughes D, Jones SA, Lin SP, Mengel E, Scarpa M, Valayannopoulos V, Giugliani R; STRIVE Investigators; Slasor P, Lounsbury D, Dummer W. Hendriksz CJ, et al. Among authors: hughes d. J Inherit Metab Dis. 2014 Nov;37(6):979-90. doi: 10.1007/s10545-014-9715-6. Epub 2014 May 9. J Inherit Metab Dis. 2014. PMID: 24810369 Free PMC article. Clinical Trial.
Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase.
Zimran A, Pastores GM, Tylki-Szymanska A, Hughes DA, Elstein D, Mardach R, Eng C, Smith L, Heisel-Kurth M, Charrow J, Harmatz P, Fernhoff P, Rhead W, Longo N, Giraldo P, Ruiz JA, Zahrieh D, Crombez E, Grabowski GA. Zimran A, et al. Among authors: hughes da. Am J Hematol. 2013 Mar;88(3):172-8. doi: 10.1002/ajh.23383. Epub 2013 Jan 22. Am J Hematol. 2013. PMID: 23339116 Free PMC article. Clinical Trial.
Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial.
Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, Parini R, Hughes D, Pastores GM, Lau HA, Al-Sayed MD, Raiman J; STRIVE Investigators; Yang K, Mealiffe M, Haller C. Hendriksz CJ, et al. Among authors: hughes d. Mol Genet Metab. 2015 Feb;114(2):178-85. doi: 10.1016/j.ymgme.2014.08.012. Epub 2014 Sep 6. Mol Genet Metab. 2015. PMID: 25284089 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: hughes da. 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: hughes da. 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.
A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease.
Byrne BJ, Geberhiwot T, Barshop BA, Barohn R, Hughes D, Bratkovic D, Desnuelle C, Laforet P, Mengel E, Roberts M, Haroldsen P, Reilley K, Jayaram K, Yang K, Walsh L; POM-001/002 Investigators. Byrne BJ, et al. Among authors: hughes d. Orphanet J Rare Dis. 2017 Aug 24;12(1):144. doi: 10.1186/s13023-017-0693-2. Orphanet J Rare Dis. 2017. PMID: 28838325 Free PMC article. Clinical Trial.
3,994 results