Children's National Medical Center

Urgent Need for Pediatric-Specific Rare Disease Treatments as 30% Die Before Age Five

- A staggering 75% of rare diseases are diagnosed during childhood, with most diagnoses occurring before age two, highlighting the critical need for pediatric-focused treatment approaches. - Current mortality rates reveal that 30% of rare disease patients do not survive past age five, emphasizing the urgent need for improved therapeutic interventions for children. - BioCryst's success in revolutionizing hereditary angioedema treatment demonstrates the potential for developing effective pediatric-specific medications for rare diseases.