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News Breaks
May 27, 2014
08:40 EDTBMRNBioMarin doses first patient in Phase 3 INSPIRE trial with BMN 701
BioMarin announced that it has dosed the first patient with BMN 701 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 glucosidas, designed to target delivery to the lysosomes where the enzyme is most needed. The INSPIRE trial focuses on late onset Pompe disease an inherited condition caused by a deficiency in the lysosomal enzyme acid alpha glucosidase, which can lead to progressive weakening of the muscles of the body, including the diaphragm, a crucial respiratory muscle. Respiratory impairment is the leading cause of morbidity and mortality in late onset Pompe disease.
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October 21, 2014
09:48 EDTBMRNShire could target BioMarin, TheStreet's Feuerstein says
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October 20, 2014
07:10 EDTBMRNAmerican Society of Human Genetics to hold annual meeting
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