Retrophin $3 million Commitment to Establish Rare Disease Network at Children’s National

Rare Daily Staff

Children’s National Health System said it was establishing the Retrophin Rare Disease Network at Children’s National with a $3 million commitment over six years to support the work of the Children’s National Rare Disease Institute, a first-of-its-kind center focused exclusively on advancing the care and treatment of children and adults with rare genetic diseases.

The Retrophin Rare Disease Network will provide funding for new dedicated positions at the CNRDI and build on the Institute’s existing digital and telemedicine programs, to extend the reach of its researchers and caregivers in areas where there is currently limited care available for children and adults living with rare diseases.

 “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, director of the CNRDI. He said the Retrophin gift will help the institute accelerate work to bring rare disease care and innovation to patients and their families.

The Retrophin Rare Disease Network will advance the CNRDI’s efforts to create a global “hub and spoke” model for disseminating and streamlining patient access to optimal care methods among national and international peer institutions. The network is designed to enhance the field of rare disease medicine by standardizing care models and establishing world-wide best practices in diagnosis and treatment.

“The tremendous difficulty that patients with rare diseases have in getting an accurate diagnosis and early treatment is well documented,” Stephen Aselage, chief executive officer of Retrophin. “The creation of this network is critical to broadening access to the Institute’s innovative resources and programs, and will ultimately lead to expanded specialized care for patients and families in the rare disease community.”

The Retrophin Rare Disease Network will provide funding for new dedicated positions at the CNRDI and build on the Institute’s existing digital and telemedicine programs, to extend the reach of its researchers and caregivers in areas where there is currently limited care available for children and adults living with rare diseases.

January 10, 2018

Photo: Stephen Aselage, CEO of Retrophin