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RAREDISEASES BIGIMPACT
RARE DISEASES WORLDWIDE
7,000
Source: National Institutes of Health
RAREDISEASES BIGIMPACT
<200,000patientsintheUnitedStates
BYDEFINITION,ARAREDISEASEAFFECTS
Source: National Institutes of Health
RAREDISEASES BIGIMPACT
30MILLION
1IN10AMERICANS
INTOTAL,RAREDISEASEIMPACT
Source: National Institutes of Health
RESEARCH PROGRESS HOPE
RAREDISEASES BIGIMPACT
1983
ORPH
AN DRUGACT
2,900ORPHANDRUG
DESIGNATIONS
SINCE1983,NEARLY
Source: Office of Orphan Products Development,
Food and Drug Administration
RAREDISEASES BIGIMPACT
AGOODSTART,but
400FDA TREATMENTS
APPROVED
<5% RAREDISEASES
HAVEATREATMENT
of
Source: Food and Drug Administration,
EveryLife Foundation for Rare Diseases
RAREDISEASES BIGIMPACTRAREDISEASES BIGIMPACT
medicines
&vaccinesin
development
452
Source: PhRMA,2013 Rare Diseases Report
RAREDISEASES BIGIMPACT
HOPE
PROGRESS
RESEARCH
Source: PhRMA

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