Congenital Adrenal Hyperplasia (CAH) is a genetic disorder affecting the adrenal glands and resulting in an impaired production of cortisol and often aldosterone. While there may not be companies exclusively focused on CAH due to its relatively rare nature, pharmaceutical companies, research institutions, and healthcare organizations may be involved in the development of treatments and support for individuals with CAH.
Neurocrine Biosciences Phase 3 results for NBT274, a potential first-line oral glucocorticoid replacement therapy for patients with classic CAH, were recently presented at the 2023 Endocrine Society's Annual Meeting. Results showed NBT274 effectively controlled hyperandrogenism with fewer side effects compared to standard treatment.
Millendo Therapeutics Announced positive preliminary data from their Phase 1b/2a clinical trial of MTL-1193, a novel once-daily oral mineralocorticoid receptor agonist for CAH. The study demonstrated good safety and tolerability, paving the way for further development.
Spruce Biosciences Achieved positive interim results from its Phase 2 trial of SPRUCE-718, a gene therapy candidate for CAH. The trial showed SPRUCE-718 could potentially restore cortisol production and reduce dependence on glucocorticoid replacement therapy.
List of Congenital Adrenal Hyperplasia Key Companies in the Market
Congenital Adrenal Hyperplasia Market Highlights:
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