What if Evie could meet the people who developed the drug for her ultra-rare disease, infantile hypophosphatasia?
Finding a Treatment for Infantile Hypophosphatasia
Among the 25 to 35 million Americans diagnosed with a rare disease, 50% are pediatric patients. Charles River partners with nonprofits, academia, and companies like Alexion to discover novel drugs and methods of study for rare diseases. We also validate mouse models to reveal new functional and behavioral endpoints in preclinical animal models.
Alexion is a global biopharmaceutical company whose life-changing therapies serve patients and families affected by rare diseases. Specializing in innovation, development, and commercialization, Alexion partners with us for safety assessment, clinical laboratory, and manufacturing support.
Asfotase alfa is an enzyme replacement therapy approved to treat patients like Evie with juvenile-onset or perinatal/infantile hypophosphotasia (HPP). It works by restoring the alkaline phosphotase (ALP) enzyme in order to promote and improve bone health. Asfotase alfa was brought to market in October 2015 after being granted Breakthrough Therapy, Priority Review, and Orphan Drug designations by the U.S. Food and Drug Administration.
Meet Ginger
Ginger was a professional singer, but everything changed the day she was diagnosed with Stage IV non-small cell lung cancer. Discover how she got her voice back.
Ginger's Story