Viral gene therapy not long-term solution for CAH

According to a study, published inHuman Gene Therapy, viral gene therapy for congenital adrenal hyperplasia provides only temporary relief, and abnormal adrenal cells will return. News Medical
Source: Society for Endocrinology - Category: Endocrinology Source Type: news

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CONCLUSIONS: The relationship between mineralocorticoid dose and PRC is complex and this may reflect variability in sampling with respect to posture, timing of last mineralocorticoid dose, adherence and concomitant medications. Our data suggests that mineralocorticoid titration should not primarily be based only on PRC normalization, but also on clinical parameters as BP and electrolyte concentration. PMID: 31613957 [PubMed - as supplied by publisher]
Source: The Journal of Clinical Endocrinology and Metabolism - Category: Endocrinology Authors: Tags: J Clin Endocrinol Metab Source Type: research
Journal Name: Journal of Pediatric Endocrinology and Metabolism Issue: Ahead of print
Source: Journal of Pediatric Endocrinology and Metabolism - Category: Endocrinology Source Type: research
Conclusion: NCCAH diagnosed in childhood, whether treated or untreated, does not pose an increased risk of overweight, obesity, or metabolic derangements in adolescence and early adulthood.
Source: Frontiers in Endocrinology - Category: Endocrinology Source Type: research
The impact of having a child with atypical genitalia due to a life-threating chronic medical condition like congenital adrenal hyperplasia (CAH) is poorly understood. Our aim was to determine parental stress and impact of CAH on parental decisions, including decisions regarding female genital restoration surgery (FGRS).
Source: Journal of Pediatric Urology - Category: Urology & Nephrology Authors: Source Type: research
Patients with congenital adrenal hyperplasia who received nonsteroidal therapy had significant reductions in androgen levels, according to results from a phase 2a study.Healio
Source: Society for Endocrinology - Category: Endocrinology Source Type: news
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Source: British Journal of Neurosurgery - Category: Neurosurgery Authors: Source Type: research
We report two cases to emphasize the importance of establishing a prompt diagnosis of CAH in male neonates, presenting with a clinical picture initially suggestive of neonatal sepsis. Inadequate response owing to delayed diagnosis in these cases may result in rapid deterioration and increased morbidity and mortality. PMID: 31584344 [PubMed - as supplied by publisher]
Source: Tropical Doctor - Category: Tropical Medicine Authors: Tags: Trop Doct Source Type: research
Journal Name: Journal of Pediatric Endocrinology and Metabolism Issue: Ahead of print
Source: Journal of Pediatric Endocrinology and Metabolism - Category: Endocrinology Source Type: research
This study confirms that CYP21A2 genotyping with next-generation sequencing and MLPA can accurately and reliably confirm the diagnosis of 21OHD. We propose a new classification by dividing group A into two new subgroups to better predict the phenotype. In light of this very high genotype-phenotype correlation, with their ever-increasing availability, declining cost, and turnaround time, we propose that molecular genetic studies can be more economical and practical alternative to the current initial diagnostic laboratory studies based on assays of intermediary steroid metabolites. PMID: 31586465 [PubMed - as supplied by publisher]
Source: European Journal of Medical Genetics - Category: Genetics & Stem Cells Authors: Tags: Eur J Med Genet Source Type: research
Non-classical congenital adrenal hyperplasia (NC-CAH) represents mild form of CAH with the prevalence of 0. 6 to 9% in women with androgen excess. Clinical and hormonal findings in females with NC-CAH are overlapping with other hyperandrogenic entities such as polycystic ovary syndrome hence causing difficulties in diagnostic approach. Metabolic consequences in subjects with NC-CAH are relatively unknown. We are lacking longitudinal follow of these patients regarding natural course of the disease or the therapeutic effects of the different drug regiments. Patients with NC-CAH similarly to those with classical form are char...
Source: Frontiers in Endocrinology - Category: Endocrinology Source Type: research
More News: Congenital Adrenal Hyperplasia | Endocrinology | Gene Therapy | Genetics | Study