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1.
Am J Med Genet ; 31(4): 821-33, 1988 Dec.
Article in English | MEDLINE | ID: mdl-3239574

ABSTRACT

Human growth hormone (HGH) inhibition may be beneficial in Duchenne muscular dystrophy (DMD) and slow down the rate of progression of the disease. The purposes of the present investigation were: 1) to assess, through pharmacological stimuli (L-dopa test), the HGH response in untreated DMD patients, and 2) to evaluate the inhibitory effect of mazindol on HGH levels as a potential treatment for DMD. In 55 DMD patients, HGH levels were measured through the L-dopa test, and 40 received mazindol. After 1 year, there was wide variability in the individual response to mazindol. An apparent diminution in the mean HGH level was observed in the whole group of patients; this was statistically significant after 3 and 6 months but not after 9 and 12 months of treatment. The results suggest that this drug is not effective for arresting growth or inhibiting HGH secretion for a prolonged period of time.


Subject(s)
Growth Hormone/blood , Indoles/pharmacology , Mazindol/pharmacology , Muscular Dystrophies/blood , Adolescent , Child , Child, Preschool , Humans , Levodopa , Male , Mazindol/adverse effects , Mazindol/therapeutic use , Muscular Dystrophies/drug therapy
4.
Am J Med Genet ; 24(3): 549-66, 1986 Jul.
Article in English | MEDLINE | ID: mdl-3524231

ABSTRACT

A controlled, double-blind therapeutic trial with the drug mazindol, a growth hormone inhibitor, was performed in a pair of 7 1/2 year-old monozygotic twins, with Duchenne muscular dystrophy (DMD). The rationale for this trial was based on a patient (reported previously) affected simultaneously with DMD and growth hormone (GH) deficiency, who is showing a benign course of the dystrophic process and is still walking at 18 years. One of the twins received 2 mg of mazindol daily, while the other received a placebo. The assessment, repeated every 2 months, included weight and height measurements, functional and motor ability tests, ergometry and determinations of serum enzymes and GH levels. After one year of trial the code was broken and it was seen that the twin under placebo treatment was strikingly worse than his brother, the progression of whose condition was practically arrested. These results strongly suggest that treatment with a GH inhibitor is beneficial for DMD patients.


Subject(s)
Diseases in Twins , Growth Hormone/antagonists & inhibitors , Indoles/therapeutic use , Mazindol/therapeutic use , Muscular Dystrophies/drug therapy , Body Height , Body Weight , Child , Clinical Trials as Topic , Double-Blind Method , Humans , Male , Muscles/physiopathology , Muscular Dystrophies/genetics , Muscular Dystrophies/physiopathology , Twins, Monozygotic
5.
Arq. bras. endocrinol. metab ; 26(1): 29-32, 1982.
Article in English | LILACS | ID: lil-7744

ABSTRACT

Foram estudadas duas pacientes que apresentavam galactorreia-amenorreia associadas a diabetes insipidas, sem aumento de sela turcica; em uma das pacientes a pneumencefalografia demonstrou a presenca de "sela vazia". A demonstracao da deficiencia uni ou multi-hormonal hipofisaria, associada a resposta a administracao de hormonios hipotalamicos liberadores, e sugestiva de doença endocrina hipotalamica


Subject(s)
Adult , Humans , Female , Amenorrhea , Diabetes Insipidus , Galactorrhea , Sella Turcica
6.
Metabolism ; 30(10): 976-81, 1981 Oct.
Article in English | MEDLINE | ID: mdl-7024724

ABSTRACT

The pituitary gonadotropic responsiveness to acute and prolonged administration of LH/FSH-releasing hormone (LHRH) were assessed in 6 patients with untreated congenital virilizing adrenal hyperplasia (partial 21-hydroxylase deficiency). The oldest subjects had normal response in comparison to females at the midfollicular phase, to the acute infusion of 25 ug LHRH regarding both gonadotropins whereas LH secretory area was decreased during the prolonged (100 ug LHRH in 8 hours) infusion with normal FSH secretion. The two youngest subjects, with higher steroid levels in our series, were either unresponsive on both ways of testing or presented pre-pubertal response.


Subject(s)
Adrenal Hyperplasia, Congenital/blood , Androgens/blood , Estrogens/blood , Gonadotropin-Releasing Hormone , Hydroxyprogesterones/blood , Pituitary Gland/metabolism , Progesterone/blood , Virilism/blood , Adolescent , Adrenal Hyperplasia, Congenital/complications , Adult , Child , Female , Humans , Pituitary Gland/drug effects , Virilism/complications
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