Researchers at the Hospital for Sick Children (SickKids) have pioneered a groundbreaking approach to overcome the challenges of conducting randomised clinical trials in paediatric rare diseases. Utilising data from an international real-world cohort, they developed a statistical method to assess the effectiveness of treatments without traditional trials. Published in Hepatology, their study focused on Alagille syndrome (ALGS), a rare genetic disorder causing liver damage. Analyzing data from untreated ALGS patients against those treated with maralixibat, the team found a significant reduction in liver-related outcomes with the medication. This approach not only benefits ALGS patients but also lays the foundation for future trials in rare diseases, offering a viable alternative to traditional methods.
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