New Drug Offers Hope for Dwarfism

Friday, June 19, 2015

Dwarfism Treatment

A new drug called BMN 111 developed by BioMarin Pharmaceuticals has improved growth in 10 children, a breakthrough that has raised hopes for treating dwarfism. Ten children who participated in the experiment grew at an average rate of 6.1 cm or 2.4 inches per year after they were administered the drug. The growth rate prior to this was 4 cm per year.

Biomarin Pharmaceuticals said that the growth rate after administering the drug was similar to that of a normal child. Dr. William R Wilcox, professor of human genetics at Emory University termed the results promising. He however, said that the children were treated for only 6 months which doesn’t give a clear picture. The drug marketed for certain children can also increase growth rate in dwarfs up to 6 centimetres per year.

BMN 111 is aimed at treating achondroplasia, the most common form of dwarfism. According to the company 24,000 children are suffering from this condition in North America, Europe, Asia and Latin America. Achondroplasia causes disproportionate growth that can lead to complications like bowed legs, sleep apnea and spinal cord compression. These complications require surgery. However, it is too early to say whether the drug can prevent these complications. The effect of the drug disappears after one year, so it is to be seen if it can increase growth year after year.  


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