Precision medicine sponsors invest heavily to identify, educate, screen, consent, genotype, and support rare patients. In many programs, once a trial ends, that...
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UK Biobank and similar resources have made an extraordinary contribution to biomedical research, enabling important advances across genomics, population health,...
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Recruitment in precision medicine and rare disease trials remains constrained by fragmented data, low prevalence populations, and heavy reliance on site-based p...
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On the latest episode of The Genetics Podcast, Patrick Short speaks with Dr. Suzanne Schindler associate professor of neurology at Washington University in St. ...
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Rare disease exposes the limits of current clinical development models. Small, fragmented populations make patient identification difficult. Heterogeneous biolo...
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Rare disease drug development involves decision-making under extreme uncertainty. Teams are asked to design trials without well-established endpoints, in small ...
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Liver disease remains one of the few major disease areas where outcomes have not improved in line with other fields such as cardiovascular disease and cancer. I...
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In rare disease and genetically stratified trials, recruitment often depends on a single critical step: confirming that a patient carries the relevant genetic v...
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Genetic testing has become a core component of patient identification and stratification in modern clinical trials, particularly in rare disease and precision m...
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On the latest episode of The Genetics Podcast, Patrick welcomed Dr. Andrea Ganna, an Associate Professor at the Institute for Molecular Medicine Finland (FIMM),...
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