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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

“Rare disease clinical trials are complex due to the additional scientific, medical, operational and regulatory requirements of newly emerging advanced therapies, such as gene therapy,” says Dr. Terence Eagleton, MB BS, Senior Medical Director at the global clinical research organization (CRO) Medpace.

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Biorepositories as a Guiding Resource for Research & Drug Discovery

XTalks

Establishing national and international research cohorts through partnerships and collaborations between teaching hospitals and biobanks is key to driving successful research programs. Dr. Bernard’s clinical research interests are in the areas of sepsis and acute respiratory distress syndrome (ARDS).

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Navigating Neuroscience Trials: Biomarkers, Imaging and CRO Strategies

XTalks

To accurately identify these genetic factors, genotyping techniques such as Sanger sequencing, next-generation sequencing (NGS) and copy number variant analysis are used. These sophisticated methods also serve as a crucial step in screening participants for study inclusion in neuroscience clinical trials.

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