Neurocrine Biosciences, Inc. (Nasdaq: NBIX) has unveiled the launch of WHAT THE C@H?!, an educational initiative designed to address the information gap surrounding congenital adrenal hyperplasia (CAH) and acknowledge the challenges faced by the community in managing this condition.
“We are proud to introduce this effort to recognize the significant physical and emotional burden individuals and their families experience living with CAH,” stated Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. “We hope this educational initiative, which was informed by insights from the community, is helpful to those navigating and managing this difficult condition.”
WHAT THE C@H?! is tailored for adults and adolescents with CAH, parents of children with CAH, and healthcare providers treating the condition. Visitors to the website can share their challenging experiences with CAH through a submission form, access educational resources, and engage with others by sharing their stories.
CAH is a rare genetic condition resulting in an enzyme deficiency affecting the production of adrenal hormones crucial for life. Individuals with CAH face the challenge of balancing cortisol deficiency and androgen excess through glucocorticoid treatment, which has been the standard for over seven decades. However, distinguishing treatment side effects from CAH symptoms poses a significant challenge in determining appropriate management strategies across different life stages.
“For over 70 years, treating the symptoms of CAH has presented challenges not only for people living with the condition and their families but also for healthcare providers,” remarked Dimitri E. Grigoriadis, Ph.D., Distinguished Scholar at Neurocrine Biosciences. “Neurocrine has a deep history in advancing research in CAH and is proud to launch this educational initiative as part of our ongoing commitment to supporting the community.”
Dina Matos, Executive Director at CARES Foundation, expressed support for WHAT THE C@H?!, highlighting its empowering and supportive platform for individuals and families affected by CAH.
The initiative includes interactive educational resources for healthcare providers, focusing on current CAH monitoring and management practices, along with insights into the potential role of corticotropin-releasing factor (CRF1) receptors in CAH.
The WHAT THE C@H?! initiative was informed by extensive interviews with individuals living with CAH, their families, and healthcare providers, as well as comprehensive market research involving 63 patients and 124 healthcare providers.
For further details and to share experiences with CAH, visit WhatTheCAH.com and follow the initiative on Facebook: What The CAH?!.
Congenital adrenal hyperplasia (CAH) is a rare genetic condition resulting in an enzyme deficiency affecting the production of adrenal hormones crucial for life. Severe enzyme deficiency can lead to adrenal crisis if left untreated, posing serious health risks including salt wasting and dehydration.
Glucocorticoids (GCs) are the current standard of care for CAH treatment, but their supraphysiologic doses may lead to significant complications such as metabolic issues, cardiovascular disease, and osteoporosis. Androgen excess associated with CAH can also lead to various health problems, underscoring the need for effective management strategies.
Corticotropin-releasing factor (CRF) regulates adrenocorticotropic hormone (ACTH) secretion, playing a crucial role in the stress response. In CAH cases caused by 21-hydroxylase enzyme deficiency, the lack of cortisol production disrupts the normal feedback loop regulating CRF secretion, contributing to adrenal androgen overproduction.
