Question

a. What is definition of rare or orphan disease? b. How many rare (orphan) diseases are there? c. How many Americans suffer from rare diseases? d. How many treatments are being studied for rare disorders?

Answer 1

Ang © Definikon : Rare or orphan disease 18 defined as any disease or condition that affects fewer than 200,000 people in the United states. i in 15 oo people. It is life threatening of chronically debilitating disease , having low prevalence .. • To be specific Rare dorease - any disease that ajecko o small percentage of the population, orphan disease - is a rare disease with lack of market large enough > 0 0 4uppet , guะใน for finding treatments for it 6 - lack of resources - lack of treatment availability .- severity of disease Ta s are. Zoophan diseases certain infectious These include diseases, birth defecte cancers ele. l conditions also lystic fibrosit, wilson's diícase etc. g5-30 million million Americane Americans wner from supper from rare diseases sao 7000 varese ona lenchie o - Researchers are doing their best to diagnose, treat & prevent rare diseases. * There is no cure for most rare diseases but appropriate treatment / care can improve quality of life & increase life expectancy. * There are oophan drugs, to treat but they are so rare would not be profitable without government help ☆ New data & genetics technologies have Ist . transformational new treatments for en rare disease National Centre for Advancing Translational suencer & Rare Diseases Research center. aimed at catalysing rare disease research. • But there in skill to alot to be done.