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Master protocol trials could reimagine the future of rare disease research

Researchers from Kennedy Krieger Institute, University of Rochester Medicine and five other institutions released a new publication detailing how master protocol trials could change the future of rare disease research.

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Researchers from Kennedy Krieger Institute, University of Rochester Medicine, and five other institutions published a study on master protocol trials for rare disease research. The paper argues that using these trials could lower costs, increase testing efficiency and capacity, and speed the delivery of treatments to patients with rare conditions. The authors suggest that a single trial framework could accommodate multiple diseases and therapies, potentially transforming how rare disease studies are conducted.

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