|
KMID : 1146120170030020041
|
|
Journal of Mucopolysaccharidosis and Rare Diseases
2017 Volume.3 No. 2 p.41 ~ p.43
|
|
|
Beneficial Effects of Growth Hormone Treatment in Prader-Willi Syndrome
|
|
Kim Jin-Sup
Yang A-Ram
Cho Sung-Yoon
Jin Dong-Kyu
|
|
|
Abstract
|
|
|
|
Prader-Willi syndrome (PWS) is a genetic disorder that is considered, especially on child, to cause poor feeding, hypotonia, failure to thrive, developmental delay and hypogonadism which is known to affect between 1 in 10,000 and 30,000 people. The children with PWS are viewed as affected by growth hormone (GH) insufficiency, although the exact mechanisms of GH deficiency are not fully understood. However, the benefits of GH treatment in children with PWS are well established. Myers, et al. (2006), Grugni, et al. (2016) indicated its positive effects on linear growth, body composition, motor function, respiratory function and psychomotor development. Despite of its effectiveness and advantages had been well known and proven in many other studies, there is only one recombinant GH product that is approved for PWS in Korea, Genotropin(R), till now. A phase III clinical study of GH treatment with EutropinTM, in 34 Korean PWS children is in progress, which is expected to have comparable effects and safety profile with the active control by assessing auxological changes such as height standard deviation score, body composition changes such as lean body mass and percent body fat, motor and cognitive development using Bayley scale, and safety profiles.
|
|
|
KEYWORD
|
|
|
Prader-Willi syndrome, Growth hormone, Clinical trial
|
|
|
FullTexts / Linksout information
|
|
|
|
|
|
Listed journal information
|
|
|
|
|