IIT-Madras Researchers Develop Drug For Rare Genetic Disorder

Researchers develop drug for a rare genetic disorder called cystinosis. 19 such cases have been reported in India
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Photo for representational purposes. (Photo: Facebook/IIT Madras)
Photo for representational purposes. (Photo: Facebook/<a href="https://www.facebook.com/ReachIITM/photos_stream">IIT Madras</a>)
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Researchers in IIT Madras have collaborated with the Cystinosis Foundation to make a wonder drug named Cysteamine Bitartrate for the rare genetic condition – cystinosis.

Children who suffer from this disorder have kidney and liver failure, growth retardation and bone deformities among other problems.

Presently in India, 19 such cases have been registered of which only 11 afflicted children are alive as reported by The Times of India.

A professor in the chemistry department told The Times of India

We began developing the drug a year back.It will be ready in another one or two months’ time.
–S Sankaraman, Professor, IIT Madras

As of now there is no known cure but once the drug is fully developed, the team hopes it will help contain the disorder.

The drug is currently only manufactured by Orphan Europe, a firm headquartered in Paris. Parents in India import the drug once in every three months from there.

The team of researches hope to market this drug at an affordable cost in India.

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