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  • Regulatory NewsRegulatory News

    Epidermolysis Bullosa: FDA Seeks to Help Development of New Treatments

    The US Food and Drug Administration (FDA) recently released guidance to help companies develop new treatments for epidermolysis bullosa (EB), which is a debilitating and often fatal rare disease. The six-page draft guidance offers details on considerations for clinical trial design, including trial population (estimates suggest 25,000-50,000 people in the US have EB), discussion of efficacy endpoints (i.e. effects on patients’ signs or symptoms such as itching, pain, bl...