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Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients.

• Authors : Pena, Loren¹; Baron, Richard²; Byrne, Barry³

Subjects: *THERAPEUTIC use of enzymes; *GLYCOGEN storage disease type II; *GENETIC disorders

• Source: Molecular Genetics & Metabolism. Feb2016, Vol. 117 Issue 2, pS92-S92. 1p.

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hCOX18 and hCOX19: Two human genes involved in cytochrome c oxidase assembly

• Authors : Sacconi, Sabrina¹; Trevisson, Eva²; Pistollato, Francesca²

Subjects: *GENES; *GENETIC mutation; *SACCHAROMYCES cerevisiae

• Source: Biochemical & Biophysical Research Communications. Nov2005, Vol. 337 Issue 3, p832-839. 8p.

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Preliminary clinical efficacy and safety of BMN 701, GILT-tagged recombinant human acid alpha glucosidase (rhGAA), in late-onset Pompe disease: results of an extension study.

• Authors : Byrne, Barry¹; Barohn, Richard²; Barshop, Bruce³

• Source: Molecular Genetics & Metabolism. Feb2014, Vol. 111 Issue 2, pS29-S29. 1p.

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POM-001 phase 1/2 study of BMN 701, GILT-tagged recombinant human (rh) GAA in late-onset Pompe disease: Initial experience in 22 patients

• Authors : Byrne, Barry¹; Barohn, Richard²; Barshop, Bruce³

• Source: Molecular Genetics & Metabolism. Feb2013, Vol. 108 Issue 2, pS28-S28. 1p.

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