Experimental Drug Hopes to Become First Treatment for Dwarfism

Published By : 18 Jun 2015 | Published By : QYRESEARCH

An experimental drug improved the level of growth on children by a considerable amount, as found in a preliminary study. The drug, developed by BioMarin Pharmaceutical is looking to become the very first approved treatment for dwarfism. The findings of a study were released on Wednesday.

According to the study, the 10 kids who received the highest dosage of the drug grew at an average rate of 2.4 inches or 6.1 centimeters every year, which is an approximately 50 per cent rise from the four centimeters per year they recorded as growing before being given the experimental drug. While on the drug, the growth rate was similar to that of a kid who does not have the condition, BioMarin Pharmaceutical said.

Analysts and investors at the Wall Street had been counting on a 50 per cent increase in growth rate and shares of the San Rafael, California based pharma company went up 6 per cent in the afterhours of trading.

Human genetics professor at Emory University in Georgia Dr William R Wilcox termed the results as “promising”. However, Dr William Wilcox expressed caution, considering only 10 kids were being administered with high dose of the drug and these children were treated for only six months. Dr Wilcox has been a consultant to BioMarin Pharmaceutical and said that even though the results in six months’ time are remarkable and important, they do not give a clear picture of the entire situation.

Human growth hormone is another drug that is already being marketed for certain kids. Dr Wilcox said that this drug can also improve the growth rate of people with dwarfism by up to six centimeters every year. However, these effects wear off after a year. It is most vital, he said, to ascertain whether the drug by BioMarin Pharmaceutical will be able to increase the growth rate of people with dwarfism year after year.
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