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How ‘rare’ is a rare disease? There is no universal or standard definition of rare disease. A disease that occurs infrequently is generally considered a rare disease. There are between 7000 - 8000 rare diseases, but less than 5% have therapies available to treat them1. Many of these diseases are genetic in origin and therefore have to be managed throughout a person’s life.

Globally, we have developed a portfolio of more than 20 medicines to treat various rare diseases in the areas of hematology and inherited metabolic disorders, including serious, life-long illnesses. There is also a growing focus on gene therapy where our dedicated research unit is working on bringing breakthrough therapies for patients.​​​​​​​


1. Ministry of Health and family Welfare, National Policy for Rare Disease, 2021. MHFW, India; 2021.

Disclaimer: This information—including product information—is intended only for residents of India. The products discussed herein may have different labelling in different countries. Patients should always ask their doctors for medical advice.PP-CPF-IND-0035 | 11/11/21
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