The $3 million pledge will support the work of the Rare Disease Institute at Children’s National for the next six years. In particular, the Retrophin Rare Disease Network will create a model to streamline patient access to optimal care, standardize care models and establish worldwide best practices in diagnosis and treatment.
“One of the chief challenges of 21st century pediatric medicine is our continued inability to provide more help to those born with rare genetic diseases,” said Marshall Summar, MD, director of the Rare Disease Institute at Children’s National. “We are grateful for this gift from Retrophin that will help us accelerate progress for our patients and families and pursue work that will have a far-reaching impact on both children and adults across the country and around the world. This is an exciting first step toward a new era of rare disease care and innovation.”
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