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Drug being developed by IIT-M for rare genetic disorder

Posted on: 27/Aug/2015 5:00:00 PM
Nearly 11 out of the 19 children who suffer out of Cystinosis are alive. IIT Madras researchers are developing a drug which can help these children from such a genetic disorder which is too rare. On the whole, there are 19 such children suffering out of Cystinosis. Parents of these children will be verily relieved and feel relaxed if this drug becomes a success and marketed in the country. Currently, the drug is being imported from Europe which costs so high as this has not got approval in our country.

Cystinosis retards the growth in children and leads to bone deformities such as rickets, blindness, etc. So far, the total diagnosed count is 19 out of which 9 are no more!

The drug called Cysteamine Bitartrate which will help treat the disorder is being made by IIT-M in collaboration with Cystinosis Foundation. The drug is at its final stage of make.

S. Sankaraman, the professor in Chemistry said they are at the final stage of purifying the compound used. The drug preparation started almost one year back. It will take two or three more months to get it ready. The drug is currently being manufactured by Orphan Europe which has its headquarters in Paris. The drug is getting imported from here in every once in three months.

Amino acid and cysteine gets accumulated in the cells. The accumulations are removed by the drug, says Nephrologist Dr Rajan Ravichandran. The drug has to be fed for every once in six hours. This can contain the disorder, but cannot cure it. For a treatment course of three months, the drug cost is about Rs 2 lakh and the drug also has customs duty of 10 to 15 percent, he added.