Treatment of partial growth hormone insensitivity syndrome

A - Human Necessities – 61 – K

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A61K 38/30 (2006.01) A61K 38/27 (2006.01) C07K 14/61 (2006.01) C07K 14/72 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2187274

Methods for increasing the growth rate of a human patient having partial growth hormone insensitivity syndrome, but not Laron syndrome, are described. One such method comprises administering an effective dose of growth hormone, preferably growth hormone with a native human sequence, with or without an N-terminal methionine, to the patient. The patient is characterized as having a height of less than about -2 standard deviations below normal for age and sex, a serum level of high-affinity growth hormone binding protein that is at least a standard deviations below normal levels, a serum level of IGF-I that is below normal mean levels, and a serum level of growth hormone that is at least normal. In another such method, the same patient population is treated with an effective amount of IGF-I, given alone or in combination with an amount of growth hormone that is effective in combination with the IGF-I.

Méthode d'accroissement de la vitesse de croissance d'un patient atteint du syndrome d'insensibilité partielle à l'hormone de croissance (différant du syndrome de Laron), consistant à lui administrer une dose efficace d'hormone de croissance (de préférence à séquence humaine native) avec ou sans méthionine en extrémité N-terminale. Le patient présente une taille inférieure à environ - 2 écarts type au-dessous de la norme correspondant à son âge et à son sexe, un taux sérique de protéine à forte affinité fixant l'hormone de croissance égal à au moins deux écarts type au-dessous de la norme correspondant à son âge et à son sexe, un taux sérique d'IGF-I inférieur aux taux moyens normaux, et un taux sérique d'hormone de croissance au moins normal. Dans une méthode voisine, la même population de patients est traitée par une dose efficace d'IGF-I administrée seule, ou avec une dose d'hormone de croissance rendue efficace par son association avec l'IGF-I.

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