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DISEASES
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» Growth Hormone Deficiency (2005)
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Biotechnology for
Growth Hormone Deficiency (2005)
The disease
Deficiency in growth hormone has very significant consequences for both adults and children. The deficiency can be acquired, as the result of a lesion (cyst, tumour, trauma, irradiation), or congenital, following mutations or a problem during foetal development.
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How many people are affected ?
Lack of growth due to a deficiency in growth hormone and Prader-Willi syndrome affect 1 live birth in 10,000 per annum. Turner’s syndrome is more common, with 1 birth in 2,000 affected. Deficiency in adults is rarer.
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How many people die from the disease?
Currently updated...
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Living with the disease
Children who have a growth hormone deficiency have an abnormally slow rate of growth and muscle mass is considerably reduced. A growth hormone deficiency can have a number of effects in adults: accumulation of fat mass, an increase in cholesterol levels causing an increase in the risk of cardiovascular accidents, a decrease in bone density, disturbance in sexual function. A number of genetic diseases (Turner’s Syndrome, Prader-Willi syndrome) are also sensitive to growth hormone. These diseases are very disabling (shortness of stature but also mental retardation and obesity).
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Treatment
Témoignage patient de
Traditional approach
The first growth hormone-based drugs appeared in the 1950’s in the United States, and in 1970 in Europe. This growth hormone was obtained by extraction from the pituitary glands of human cadavers. In addition to the difficulty of supply (resulting in limited use), the dramatic side effect of this pituitary-derived hormone was infection of patients with Creuzfeld-Jacob disease. More than a hundred cases occurred in France.
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Biotech revolutions
At the end of the 1980’s, patients were offered a synthetic human growth hormone produced using the techniques of biotechnology which carried no risk of infection. Another “biodrug” was developed to stimulate endogenous production of the hormone by the patient. The large scale production also permits to expand therapeutic indication to more patients (especialy to patients with orphean diseases)
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Drugs currently available
Two recombinant proteins have been developed: somatropin, which is growth hormone itself and sermorelin, a hormone which stimulates production of growth hormone. These proteins are then introduced into medical preparations put on the market by a number of different pharmaceutical companies.
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Number of patients treated
In France, the number of children and adults treated with growth hormone is estimated to be 10,000 per annum.
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Future
The developments expected in the treatment of growth hormone deficiency are essentially a reduction in the number of injections (currently once a day) and new types of administration (inhalers, for example).
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Costs to society
For a child, at an average price of €40/mg, an average weight of 20 kg and 1 dose of 1 unit/kg/week, a year’s treatment costs €13, 000. For adults, at a dose of 0.5 mg per day, the annual cost of a substitute treatment is in the region of €7, 000.
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