Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study
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Title
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study
Authors
Keywords
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Journal
GENETICS IN MEDICINE
Volume -, Issue -, Pages -
Publisher
Springer Nature
Online
2018-03-22
DOI
10.1038/gim.2018.2
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- A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
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- Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease
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- Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease
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- Reference Curves for the Gross Motor Function Measure: Percentiles for Clinical Description and Tracking Over Time Among Children With Cerebral Palsy
- (2008) Steven E Hanna et al. PHYSICAL THERAPY
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