An ‘Adaptive Treatment Strategy’ for Oral Vancomycin in Patients with the Orphan Disease Primary Sclerosing Cholangitis

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An ‘Adaptive Treatment Strategy’ for Oral Vancomycin in Patients with the Orphan Disease Primary Sclerosing Cholangitis

This article discusses the benefits of therapeutic decision-making in the context of orphan diseases, focusing on PSC as an example. It also proposes an adaptive treatment strategy to trial oral vancomycin therapy in PSC patients with or without ulcerative colitis.

This article brings hope to the PSC community by proposing possible alternate avenues that patients can access oral vancomycin—and ways other rare disease patients can access therapies too.

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