Format
Sort by
Items per page

Send to

Choose Destination

Links from PubMed

Items: 1 to 20 of 107

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.

J Inherit Metab Dis. 2014 Nov;37(6):979-90. doi: 10.1007/s10545-014-9715-6. Epub 2014 May 9.

2.

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.

Mol Genet Metab. 2015 Feb;114(2):178-85. doi: 10.1016/j.ymgme.2014.08.012. Epub 2014 Sep 6.

3.

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.

Mol Genet Metab. 2016 Sep;119(1-2):131-43. doi: 10.1016/j.ymgme.2016.05.018. Epub 2016 Jun 16.

4.

Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study.

Long B, Tompkins T, Decker C, Jesaitis L, Khan S, Slasor P, Harmatz P, O'Neill CA, Schweighardt B.

Clin Ther. 2017 Jan;39(1):118-129.e3. doi: 10.1016/j.clinthera.2016.11.017. Epub 2016 Dec 10.

5.

Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study.

Burton BK, Berger KI, Lewis GD, Tarnopolsky M, Treadwell M, Mitchell JJ, Muschol N, Jones SA, Sutton VR, Pastores GM, Lau H, Sparkes R, Genter F, Shaywitz AJ, Harmatz P.

Am J Med Genet A. 2015 Oct;167A(10):2272-81. doi: 10.1002/ajmg.a.37172. Epub 2015 Jun 10.

6.

Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial.

Schweighardt B, Tompkins T, Lau K, Jesaitis L, Qi Y, Musson DG, Farmer P, Haller C, Shaywitz AJ, Yang K, O'Neill CA.

Clin Ther. 2015 May 1;37(5):1012-1021.e6. doi: 10.1016/j.clinthera.2014.11.005. Epub 2014 Dec 6.

PMID:
25487082
7.

Elosulfase Alfa: a review of its use in patients with mucopolysaccharidosis type IVA (Morquio A syndrome).

Lyseng-Williamson KA.

BioDrugs. 2014 Oct;28(5):465-75. doi: 10.1007/s40259-014-0108-z. Review.

PMID:
25200032
8.

Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study.

Harmatz PR, Mengel E, Geberhiwot T, Muschol N, Hendriksz CJ, Burton BK, Jameson E, Berger KI, Jester A, Treadwell M, Sisic Z, Decker C.

Am J Med Genet A. 2017 Feb;173(2):375-383. doi: 10.1002/ajmg.a.38014. Epub 2016 Oct 24.

9.

Pharmacokinetic and pharmacodynamic evaluation of elosulfase alfa, an enzyme replacement therapy in patients with Morquio A syndrome.

Qi Y, Musson DG, Schweighardt B, Tompkins T, Jesaitis L, Shaywitz AJ, Yang K, O'Neill CA.

Clin Pharmacokinet. 2014 Dec;53(12):1137-47. doi: 10.1007/s40262-014-0173-y.

10.

Elosulfase alfa (BMN 110) for the treatment of mucopolysaccharidosis IVA (Morquio A Syndrome).

Hendriksz CJ.

Expert Rev Clin Pharmacol. 2016 Dec;9(12):1521-1532. Epub 2016 Nov 23. Review.

PMID:
27855521
11.

Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y.

Jones SA, Bialer M, Parini R, Martin K, Wang H, Yang K, Shaywitz AJ, Harmatz P.

Pediatr Res. 2015 Dec;78(6):717-22. doi: 10.1038/pr.2015.169. Epub 2015 Sep 2.

12.

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.

J Inherit Metab Dis. 2016 Nov;39(6):839-847. Epub 2016 Aug 23.

13.

Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa.

Hughes D, Giugliani R, Guffon N, Jones SA, Mengel KE, Parini R, Matousek R, Hawley SM, Quartel A.

Orphanet J Rare Dis. 2017 May 23;12(1):98. doi: 10.1186/s13023-017-0634-0.

14.

Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations.

Tomatsu S, Sawamoto K, Shimada T, Bober MB, Kubaski F, Yasuda E, Mason RW, Khan S, Alméciga-Díaz CJ, Barrera LA, Mackenzie WG, Orii T.

Expert Opin Orphan Drugs. 2015 Nov 1;3(11):1279-1290. Epub 2015 Oct 29.

15.

Elosulfase alfa.

Haddley K.

Drugs Today (Barc). 2014 Jul;50(7):475-83. doi: 10.1358/dot.2014.50.7.2177904. Review.

PMID:
25101330
16.

Elosulfase alfa: first global approval.

Sanford M, Lo JH.

Drugs. 2014 Apr;74(6):713-8. doi: 10.1007/s40265-014-0210-z.

PMID:
24700469
17.

[Morquio disease (Mucopolysaccharidosis type IV-A): clinical aspects, diagnosis and new treatment with enzyme replacement therapy].

Politei J, Schenone AB, Guelbert N, Fainboim A, Szlago M.

Arch Argent Pediatr. 2015 Aug;113(4):359-64. doi: 10.5546/aap.2015.359. Review. Spanish.

18.

Antibodies that neutralize cellular uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndrome.

Melton AC, Soon RK Jr, Tompkins T, Long B, Schweighardt B, Qi Y, Vitelli C, Bagri A, Decker C, O'Neill CA, Zoog SJ, Jesaitis L.

J Immunol Methods. 2017 Jan;440:41-51. doi: 10.1016/j.jim.2016.10.006. Epub 2016 Oct 24.

19.

Elosulfase alfa enzyme replacement therapy attenuates disease progression in a non-ambulatory Japanese patient with Morquio A syndrome (case report).

Hiramatsu M, Nakamura K.

Mol Genet Metab Rep. 2017 Sep 14;13:76-79. doi: 10.1016/j.ymgmr.2017.09.001. eCollection 2017 Dec.

20.

Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome.

Harmatz PR, Mengel KE, Giugliani R, Valayannopoulos V, Lin SP, Parini R, Guffon N, Burton BK, Hendriksz CJ, Mitchell JJ, Martins AM, Jones SA, Guelbert N, Vellodi A, Wijburg FA, Yang K, Slasor P, Decker C.

Mol Genet Metab. 2015 Feb;114(2):186-94. doi: 10.1016/j.ymgme.2014.10.015. Epub 2014 Nov 1.

Supplemental Content

Support Center