dedicated to improving the health and quality of life of Americans with rare diseases by making orphan drugs available in the U.S.
Watch the four-part video series created to help patients and caregivers better understand the disease following a diagnosis, including treatment options.
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An illness that affects fewer than 200,000 Americans at any given time, thereby limiting scientific research, clinical expertise and patient access to effective treatment options
A medication or other medicinal product used to treat a rare disease or disorder