Lipoid congenital adrenal hyperplasia

Spruce Biosciences Presents Phase 2 Data for Tildacerfont in Adults with Congenital Adrenal Hyperplasia at 23rd European Congress of Endocrinology

Retrieved on: 
Monday, May 24, 2021
Health, Other Health, Clinical trials, General Health, Pharmaceutical, Biotechnology, Medicine, Medical specialties, Organ systems, Congenital adrenal hyperplasia, Adrenal gland disorders, Hyperplasia, Adrenal gland, Rare diseases, Congenital adrenal hyperplasia due to 11β-hydroxylase deficiency, Lipoid congenital adrenal hyperplasia, Tildacerfont, Spruce Biosciences, TILDACERFONT, SPRUCE BIOSCIENCES

Administration of tildacerfont to these patients did not lead to significant changes in these levels.

Key Points: 
  • Administration of tildacerfont to these patients did not lead to significant changes in these levels.
  • Tildacerfont has been evaluated in 171 patients across seven clinical trials in which it has been generally well tolerated.
  • Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet need.
  • Spruce is initially developing its wholly-owned product candidate, tildacerfont, as the potential first non-steroidal therapy for patients suffering from classic congenital adrenal hyperplasia (CAH).

Neurocrine Biosciences Reports Positive Phase II Data for Crinecerfont in Adults with Congenital Adrenal Hyperplasia at ENDO Online 2020

Retrieved on: 
Monday, June 8, 2020
Adrenal gland disorders, Endocrine diseases, Medicine, Branches of biology, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency, 21-Hydroxylase, Glucocorticoid, Adrenal gland, Congenital adrenal hyperplasia, Lipoid congenital adrenal hyperplasia

These data suggest that crinecerfont has the potential to improve CAH symptoms and to reduce the burden of daily glucocorticoid exposure for these patients.

Key Points: 
  • These data suggest that crinecerfont has the potential to improve CAH symptoms and to reduce the burden of daily glucocorticoid exposure for these patients.
  • Neurocrine Biosciences plans to initiate a single, global registrational study of crinecerfont in adult patients with classic CAH in the second half of 2020.
  • Classic CAH is a genetic disorder, in which an enzyme deficiency alters the production of adrenal steroids.
  • The Phase II open-label, multiple-dose, dose-finding study assessed the safety, tolerability, pharmacokinetics and pharmacodynamics of crinecerfont in 18 adults with classic 21-hydroxylase deficiency CAH.

Millendo Therapeutics Announces 21st European Congress of Endocrinology 2019 Presentations on Livoletide for Prader-Willi Syndrome and Nevanimibe for Classic Congenital Adrenal Hyperplasia

Retrieved on: 
Wednesday, May 8, 2019
Health, Biotechnology, Clinical trials, Pharmaceutical, Endocrine diseases, Adrenal gland disorders, Organ systems, Endocrinology, Adrenal insufficiency, Congenital adrenal hyperplasia, Adrenal gland, Cushing's syndrome, Hyperplasia, CAH, Lipoid congenital adrenal hyperplasia, Congenital adrenal hyperplasia due to 21-hydroxylase deficiency

Livoletide, a potential first-in-class treatment for Prader-Willi syndrome (PWS), will be featured in two poster presentations.

Key Points: 
  • Livoletide, a potential first-in-class treatment for Prader-Willi syndrome (PWS), will be featured in two poster presentations.
  • The company will also give an overview of its ongoing nevanimibe Phase 2b study in patients with classic congenital adrenal hyperplasia (CAH) in a third poster presentation.
  • Nevanimibe decreases adrenal steroidogenesis through the inhibition of ACAT1 and is being studied for the treatment of two orphan adrenal diseases: classic congenital adrenal hyperplasia (CAH) and endogenous Cushings syndrome (CS).
  • The company is currently advancing livoletide for the treatment of Prader-Willi syndrome and nevanimibe for the treatment of classic congenital adrenal hyperplasia and endogenous Cushings syndrome.

Congenital Adrenal Hyperplasia Epiomic Epidemiology Forecasts 2018-2028

Retrieved on: 
Monday, September 17, 2018
Adrenal gland disorders, Medicine, Endocrine system, Endocrinology, Congenital adrenal hyperplasia, Adrenal gland, Congenital adrenal hyperplasia due to 11β-hydroxylase deficiency, Mineralocorticoid, Cytochrome P450 reductase, Glucocorticoid, Steroid, Lipoid congenital adrenal hyperplasia

The "Congenital Adrenal Hyperplasia Forecast In 31 Major Markets 2018-2028" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Congenital Adrenal Hyperplasia Forecast In 31 Major Markets 2018-2028" report has been added to ResearchAndMarkets.com's offering.
  • Congenital adrenal hyperplasia (CAH) is a group of genetic disorders associated with deficiencies of several adrenal enzymes which lead to abnormal production of glucocorticoids, mineralocorticoids and sex steroids.
  • This, in turn, affects metabolism, sodium and water balance and the development of primary or secondary sex characteristics.
  • CAH can occur in classic (severe) or non-classic (mild) forms depending on the amount of residue enzyme activity.