Korean scientists have shown the effectiveness of treatment with recombinant human growth hormone for young children with Prader-Willi syndrome

Recombinant Human Growth Hormone (rhGH) preparations have been approved for use in children with Prader-Willi syndrome at the US FDA in 2000 and EMA in 2001. However, systematic studies of the treatment of infants and children under 2 years of age with this drug were not enough. Korean scientists have done new research and found that growth hormone treatment improves the growth and body composition of patients with Prader-Willi syndrome.

Prader-Willi Syndrome is a rare genetic disease. The disease occurs as a result of loss of gene expression in the chromosome 15q11-q13 inherited from the father and is characterized by muscle hypotension, developmental disability in infancy, short stature, psychomotor retardation and hyperphagia, leading to severe obesity, as well as hypothalamic dysfunction, which can manifest itself at a later age . Patients with Prader-Willi syndrome also have a special body composition with a high percentage of fat and low muscle mass. This picture is observed even in children with insufficient weight with Prader-Willi syndrome.

A study by Korean scientists aimed at confirming that Eutropin - an rhGH drug - improves body composition, growth, motor and cognitive skills.

Treatment of rhGH for 52 weeks in infants and young children with Prader-Willi syndrome improved growth, motor and cognitive development, and the results for Eutropin's efficacy and safety were comparable to those of Genotropin. Consequently, Eutropin is expected to provide safe and clinically significant improvements in children with Prader-Willi syndrome.

The study is published in the Orphanet Journal of Rare Diseases.