The FDA grants rare pediatric disease designation for diseases with serious or life- threatening conditions that affect people from birth to 18 years of age, that affect fewer than 200,000 people in the U.S.
The toolbox has been developed through funding from the EJP RD project.
The European Joint Programme on Rare Diseases is an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N°825575
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