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

The primary treatment outcomes of a phase 2, randomized, double‐blind, pilot study evaluating safety, physiological, and pharmacological effects of elosulfase alfa in patients with Morquio A syndrome are herewith presented. Patients aged ≥7 years and able to walk ≥200 m in the 6‐min walk test (6MWT)...

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Detalhes bibliográficos
Publicado no:Am J Med Genet A
Main Authors: Burton, Barbara K., Berger, Kenneth I., Lewis, Gregory D., Tarnopolsky, Mark, Treadwell, Marsha, Mitchell, John J., Muschol, Nicole, Jones, Simon A., Sutton, V. Reid, Pastores, Gregory M., Lau, Heather, Sparkes, Rebecca, Genter, Fred, Shaywitz, Adam J., Harmatz, Paul
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2015
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4744659/
https://ncbi.nlm.nih.gov/pubmed/26069231
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.a.37172
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