ORPHAN DRUG and ORPHAN DISEASE

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Offline Farhana Israt Jahan

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ORPHAN DRUG and ORPHAN DISEASE
« on: February 05, 2020, 01:36:34 PM »
ORPHAN DRUG and ORPHAN DISEASE
An orphan drug is a pharmaceutical agent that has been developed specially to treat a rare medical condition; the condition itself being referred to as an orphan disease. Orphan disease is a disease that affects a small percentage of the population.

Examples of orphan disease & drugs:
Wilson’s disease: Penicillamine was developed to treat wilson’s disease, a rare hereditary disease that can lead to a fatal accumulation of copper in the body. The drug was later found to be effective in treating arthritis.
Cystic fibrosis: Cystic fibrosis is an autosomal recessive genetic disorder affecting most critically the lungs & also the pancreas, liver & intestine. In the 1980s, cystic fibrosis patients are rarely found. Drugs like pulmozyme & tobramycin both developed that significantly improve their quality of life.
Farhana Israt Jahan
Assistant Professor
Dept. of Pharmacy

Offline mithi

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Re: ORPHAN DRUG and ORPHAN DISEASE
« Reply #1 on: May 04, 2020, 04:55:07 PM »
thanks for sharing
Faria Mannan Mithi
Lecturer, Department of Pharmacy
Faculty of Allied Health Science
Daffodil International University
E-mail:mithi.ph@diu.edu.bd