San Rafael, Calif., May 27, 2014 – BioMarin Pharmaceutical Inc. announced today that it has dosed the first patient with BMN 701 (GILT-tagged Recombinant Human GAA) in the Phase 3 INSPIRE trial for Pompe disease. BMN 701 is a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), designed to target delivery to the lysosomesA small part of the cell that acts like a recycling center, helping break down waste materials. More where the enzymeA protein that helps the body carry out chemical reactions. More is most needed. The INSPIRE trial focuses on late onset Pompe diseaseA form of Pompe disease that begins after infancy and usually progresses more slowly. More – an inherited condition caused by a deficiency in the lysosomal enzymeA protein that helps the body carry out chemical reactions. More acid alpha glucosidase, which can lead to progressive weakening of the muscles of the body, including the diaphragmThe main muscle used for breathing. More, a crucial respiratory muscle. Respiratory impairment is the leading cause of morbidity and mortality in late onset Pompe diseaseA form of Pompe disease that begins after infancy and usually progresses more slowly. More.
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