✦ LIBER ✦

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

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

Book ID: 125356708
Publisher: Springer
Year: 2014
Tongue: English
Weight: 358 KB
Volume: 37
Category: Article
ISSN: 0141-8955
DOI: 10.1007/s10545-014-9715-6

No coin nor oath required. For personal study only.

📜 SIMILAR VOLUMES

A multi-national, randomized, double-bli

A multi-national, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of BMN 110 treatment for mucopolysaccharidosis IVA (Morquio syndrome type A)

✍ Hendriksz, Christian; Burton, Barbara K.; Fleming, Thomas; Giugliani, Roberto; H 📂 Article 📅 2013 🏛 Elsevier Science 🌐 English ⚖ 65 KB

Long Term Outcomes of a Phase 1/2, Multi

Long Term Outcomes of a Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)

✍ Christian Hendriksz; Ashok Vellodi; Simon Jones; Hiwot Takkele; S. Lee; Sarah Ch 📂 Article 📅 2012 🏛 Elsevier Science 🌐 English ⚖ 57 KB

A randomised, double-blind, placebo-cont

A randomised, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of three dosing schedules of agalsidase alfa enzyme replacement therapy for Fabry disease

✍ Hughes, D.A.; Deegan, P.B.; Milligan, A.; Wright, N.; Butler, L.H.; Jacobs, A.; 📂 Article 📅 2013 🏛 Elsevier Science 🌐 English ⚖ 283 KB