PMC Links for Books (Select 4866362) - PMC

Select item 47446591.

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

Barbara K. Burton, Kenneth I. Berger, Gregory D. Lewis, Mark Tarnopolsky, Marsha Treadwell, John J. Mitchell, Nicole Muschol, Simon A. Jones, V. Reid Sutton, Gregory M. Pastores, Heather Lau, Rebecca Sparkes, Fred Genter, Adam J. Shaywitz, Paul Harmatz

Am J Med Genet A. 2015 Oct; 167(10): 2272–2281. Published online 2015 Jun 10. doi: 10.1002/ajmg.a.37172

PMCID:: PMC4744659

Article PDF–592K

Select item 45012262.

Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease

Lubor Goláň, Ozlem Goker-Alpan, Myrl Holida, Ikka Kantola, Mariusz Klopotowski, Johanna Kuusisto, Aleš Linhart, Jacek Musial, Kathleen Nicholls, Derlis Gonzalez-Rodriguez, Reena Sharma, Bojan Vujkovac, Peter Chang, Anna Wijatyk

Drug Des Devel Ther. 2015; 9: 3435–3444. Published online 2015 Jul 8. doi: 10.2147/DDDT.S80928

PMCID:: PMC4501226

Article PDF–347K

Select item 42602553.

Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study

Raphael Schiffmann, Gregory M Pastores, Yeong-Hau H Lien, Victoria Castaneda, Peter Chang, Rick Martin, Anna Wijatyk

Orphanet J Rare Dis. 2014; 9: 169. Published online 2014 Nov 26. doi: 10.1186/s13023-014-0169-6

PMCID:: PMC4260255

Article PDF–800K

Select item 42067724.

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

Christian J. Hendriksz, Barbara Burton, Thomas R. Fleming, Paul Harmatz, Derralynn Hughes, Simon A. Jones, Shuan-Pei Lin, Eugen Mengel, Maurizio Scarpa, Vassili Valayannopoulos, Roberto Giugliani, STRIVE Investigators, Peter Slasor, Debra Lounsbury, Wolfgang Dummer

J Inherit Metab Dis. 2014; 37(6): 979–990. Published online 2014 May 9. doi: 10.1007/s10545-014-9715-6

PMCID:: PMC4206772

Article PDF–355K

Select item 42037125.

Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency

Vassili Valayannopoulos, Vera Malinova, Tomas Honzík, Manisha Balwani, Catherine Breen, Patrick B. Deegan, Gregory M. Enns, Simon A. Jones, John P. Kane, Eveline O. Stock, Radhika Tripuraneni, Stephen Eckert, Eugene Schneider, Gavin Hamilton, Michael S. Middleton, Claude Sirlin, Bruce Kessler, Christopher Bourdon, Simeon A. Boyadjiev, Reena Sharma, Chris Twelves, Chester B. Whitley, Anthony G. Quinn

J Hepatol. Author manuscript; available in PMC 2015 Nov 1.

Published in final edited form as: J Hepatol. 2014 Nov; 61(5): 1135–1142. Published online 2014 Jun 30. doi: 10.1016/j.jhep.2014.06.022

PMCID:: PMC4203712

Article PDF–1.2M

Select item 41070786.

Natural History and Galsulfase Treatment in Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) — 10 Year Follow-up of Patients Who Previously Participated in an MPS VI Survey Study

Roberto Giugliani, Christina Lampe, Nathalie Guffon, David Ketteridge, Elisa Leão Teles, James E. Wraith, Simon A Jones, Cheri Piscia-Nichols, Ping Lin, Adrian Quartel, Paul Harmatz

Am J Med Genet A. Author manuscript; available in PMC 2015 Aug 1.

Published in final edited form as: Am J Med Genet A. 2014 Aug; 164(8): 1953–1964. Published online 2014 Apr 24. doi: 10.1002/ajmg.a.36584

PMCID:: PMC4107078

Article PDF–856K

Select item 40524277.

A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4–7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy

Roberto Giugliani, Wuh-Liang Hwu, Anna Tylki-Szymanska, David A.H. Whiteman, Arian Pano

Genet Med. 2014 Jun; 16(6): 435–441. Published online 2013 Nov 7. doi: 10.1038/gim.2013.162

PMCID:: PMC4052427

Article PDF–419K

Select item 37281698.

Clinical Effect and Safety Profile of Recombinant Human Lysosomal Acid Lipase in Patients with Cholesteryl Ester Storage Disease

Manisha Balwani, Catherine Breen, Gregory M Enns, Patrick B Deegan, Tomas Honzík, Simon Jones, John P Kane, Vera Malinova, Reena Sharma, Eveline O Stock, Vassili Valayannopoulos, J Edmond Wraith, Jennifer Burg, Stephen Eckert, Eugene Schneider, Anthony G Quinn

Hepatology. Author manuscript; available in PMC 2014 Sep 1.

Published in final edited form as: Hepatology. 2013 Sep; 58(3): 950–957. Published online 2013 Mar 28. doi: 10.1002/hep.26289

PMCID:: PMC3728169

Article PDF–1.5M

Select item 27473399.

Review of the safety and efficacy of imiglucerase treatment of Gaucher disease

Deborah Elstein, Ari Zimran

Biologics. 2009; 3: 407–417. Published online 2009 Sep 15. doi: 10.2147/btt.2009.3497

PMCID:: PMC2747339

Article PDF–191K

Select item 272607110.

Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID)

Claire Booth, H Bobby Gaspar

Biologics. 2009; 3: 349–358. Published online 2009 Jul 13.

PMCID:: PMC2726071

Article PDF–174K

Select item 118200911.

Long-Term Safety and Efficacy of Enzyme Replacement Therapyfor Fabry Disease

William R. Wilcox, Maryam Banikazemi, Nathalie Guffon, Stephen Waldek, Philip Lee, Gabor E. Linthorst, Robert J. Desnick, Dominique P. Germain, International Fabry Disease Study Group

Am J Hum Genet. 2004 Jul; 75(1): 65–74. Published online 2004 May 20. doi: 10.1086/422366

PMCID:: PMC1182009

Article PDF–173K

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