Survey: Help Improve Rare Disease Research

Developing quality statements for rare disease via consensus across the rare disease community
The Rare Disease Quality Statement Project is gathering insights from patients, caregivers, and researchers to improve rare disease research and support. Your feedback is invaluable in shaping better resources and outcomes.
📢 Take the survey now: RareDiseaseQS.org
Recent Blog Posts
Article: Adult Quality of Life with CAH
A new study published in Endocrine Connections explores the lived experiences of adults with congenital adrenal hyperplasia (CAH) and the wide-ranging impact the condition can have on quality of life. Through in-depth interviews with adults living with CAH as well as...
Liquid Hydrocortisone Discontinued in the UK
Liquid hydrocortisone injections have been discontinued in the UK, affecting emergency treatment for people with adrenal insufficiency. This follows the supply shortage reported in February 2025 -...
Medicine security – a national UK priority.
Concerns about medicines shortages in the UK have been highlighted in recent analysis from the House of Lords Public Services Committee and the Royal Pharmaceutical Society. The House of Lords Public Services Committee warns that medicines supply should be treated as...


