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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2015
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4744659/
https://www.ncbi.nlm.nih.gov/pubmed/26069231
http://dx.doi.org/10.1002/ajmg.a.37172

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