See How This Little Girl Changed the Future of Drug Development
Finding a Treatment for Batten Disease
Mila received her first dose of Milasen at Boston Children's Hospital on January 31, 2018, after the FDA reviewed all the data and approved Mila's trial within a week. Along with the clinical team at Boston Children's Hospital, we achieved for Mila what had never been done before: a novel drug treatment within a year of her diagnosis, and an approved personalized Batten disease treatment for her just six weeks after the first trial.
Boston Children's Hospital is ranked the number one pediatric hospital in the nation by U.S. News and World Report. It is home to the world's largest pediatric research enterprise, the leading recipient of pediatric research funding from the National Institutes of Health, and the primary pediatric teaching hospital for Harvard Medical School. Boston Children’s treats more children with rare diseases and complex conditions than any other hospital.
Charles River has several decades of IND research experience, a unique range of services, and best-in-class expertise to help clients successfully initiate and complete IND-enabling programs on time and within budget. The same experience can also be applied to the design of suitable studies or programs for submission to any global regulatory authorities. We were proud to support Dr. Yu with regulatory consulting, toxicology and behavioral testing, bioanalysis, and drug product safety testing.
Meet Evie
When Evie was born, she wasn't expected to survive due to the metabolic bone disease infantile hypophosphotasia (HPP). What if Evie could meet the people who developed the drug for her ultra-rare disease?
Evie's Story