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

Efficacy and safety of elosulfase alfa enzyme replacement therapy (ERT) were assessed in an open‐label, phase 2, multi‐national study in Morquio A patients aged ≥5 years unable to walk ≥30 meters in the 6‐min walk test. Patients received elosulfase alfa 2.0 mg/kg/week intravenously for 48 weeks. Eff...

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Dades bibliogràfiques
Publicat a:Am J Med Genet A
Autors principals: Harmatz, Paul R., Mengel, Eugen, Geberhiwot, Tarekegn, Muschol, Nicole, Hendriksz, Christian J., Burton, Barbara K., Jameson, Elisabeth, Berger, Kenneth I., Jester, Andrea, Treadwell, Marsha, Sisic, Zlatko, Decker, Celeste
Format: Artigo
Idioma:Inglês
Publicat: John Wiley and Sons Inc. 2016
Matèries:
Original Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5298029/
https://ncbi.nlm.nih.gov/pubmed/27774754
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.a.38014
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