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rare disease drug development | The Addi and Cassi Fund - Niemann Pick Type C

Tuesday, September 28, 2021

Orphan Drug Act – A Collosal Failure Considering Rare Disease Drug Statistics

May 23, 2010 by  
Filed under Featured Stories

For the past week, I have been obsessing over rare disease statistics ever since it was published that 80% of all rare diseases are ‘ultra orphans’ – affecting 6,000 people per disease state or less – and only 15% of all rare disease drug applications filed with the FDA are for ‘ultra orphans.’ Yesterday, I […]