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Translational Bytes
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Gene Therapy Grows Up
Gene therapy is almost 30 years old with only a handful of approved products across the world and none in the United States. Thus, it should come as no surprise that the history and development of gene therapy has had many twists and turns. Recently, in Clinical and Translational Science...[read more]
New Drug Rounds: Translational Biomarkers
A main goal of translational science is to understand the pathology of human disease and find treatments that have a relevant mechanism of action. In some cases, disease mechanisms may be well-understood, as for inherited disorders...[read more
Last year, the drug-development community, regulators, medical practitioners, and the public were shocked by the unanticipated serious adverse events in healthy subjects in a study of a small molecule fatty acid amide hydroxylase inhibitor BIA-102474. This tragic development precipitated...[read more
A Potential New Clinical Biomarker for Multiple Sclerosis – Quantification of Galactosylceramide
Advancements in the discovery and development of new therapeutics are often limited by the lack of robust biomarkers to assess drug activity.  In a recent paper in Clinical and Translational Science...[read more]
Awarded the “Design of the Year” by the London Design Museum in 2015, microphysiological systems or “organs-on-chips” could fundamentally change drug toxicity testing. Despite decades of research using animal and cell models, predicting drug-induced toxicity in humans...[read more
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