Safety and Efficacy of Elosulfase Alfa in Australian Patients with Morquio a Syndrome: A Phase 3b Study

Safety and Efficacy of Elosulfase Alfa in Australian Patients with Morquio a Syndrome: A Phase 3b Study

Abstract The safety and efficacy of elosulfase alfa were evaluated in a multicenter, open-label, phase 3b study in Australian Morquio A patients, consisting of a 49-week initial phase and an extension phase until elosulfase alfa was government funded. Thirteen patients (1-27 years) were enrolled. No...

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Bibliographic Details
Main Authors: Kaustuv Bhattacharya, Shanti Balasubramaniam, Kevin Murray, Heidi Peters, David Ketteridge, Anita Inwood, Joy Lee, Carolyn Ellaway, Penny Owens, Melanie Wong, Christine Ly, Jim McGill
Format: Article
Language:English
Published: SciELO 2020-05-01
Series:Journal of Inborn Errors of Metabolism and Screening
Subjects:
efficacy
elosulfase alfa
enzyme replacement therapy
mucopolysaccharidosis IVA
Morquio A syndrome
safety
Online Access:http://www.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942020000100303&tlng=en

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http://www.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942020000100303&tlng=en

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