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Clinical Trial

May 15, 2018 Amy Simmonds

A clinical trial is research designed to understand the safety and efficacy of a drug, biologic or device.  There are 4 phases to most clinical trials from Phase 1 that seeks to answer safety concerns in a healthy population of volunteers to Phase 2 trials aimed at understanding the safety of the product in a specific disease population.  Phase 3 trials further test the products efficacy and Phase 4 clinical trials capture real-world data upon approval of the product by the Food and Drug Administration (FDA).  For more on clinical trials, visit www.clinicaltrials.gov.

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A day in which each patient receives an early diagnosis, has access to state-of-the-art care, and lives a life free of Krabbe disease.

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Pennsylvania (Opt-In)

Currently Krabbe disease is not automatically screened for in the state of Pennsylvania. Parents must request or “Opt-In” to have Krabbe added to the newborn screening tests done at birth.

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