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Sangamo BioSciences, Inc. announced the presentation of new preclinical data in disease models from its In Vivo Protein Replacement Platform™ (IVPRP) programs for MPS I (Hurler syndrome) and MPS II (Hunter syndrome). The data demonstrate that the Company's IVPRP approach enabled stable production of therapeutic levels of replacement enzyme from the liver into the circulation and secondary tissues, including the brain, resulting in significant reduction in biomarkers of the disease and, importantly, statistically significant improvements in cognitive function in treated animals.
Sanofi and its specialty care global business unit Sanofi Genzyme presented data from NEO1, its Phase 1/2 clinical study evaluating the investigational novel enzyme replacement therapy neoGAA in 24 patients with late-onset Pompe disease.
Decreased opioid use prior to joint replacement surgery has been linked with improved patient satisfaction and outcomes, fewer complications and a reduced need for post-surgical opioids, according to two new studies.