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About our Rare Diseases news
Latest news on rare diseases, providing comprehensive coverage of research breakthroughs, treatment developments, patient stories, and advocacy efforts for these often-overlooked conditions.
Rare diseases, also known as orphan diseases, affect a small percentage of the population but collectively impact millions worldwide. With over 7,000 identified rare diseases, these conditions pose unique challenges for patients, healthcare providers, and researchers alike. According to the National Institutes of Health, a disease is considered rare if it affects fewer than 200,000 people in the United States.
Recent advancements in genetic research and precision medicine have led to promising developments in the diagnosis and treatment of rare diseases. The European Joint Programme on Rare Diseases (EJP RD) continues to foster collaboration among researchers, clinicians, and patient organisations to accelerate progress. Additionally, the UK Rare Diseases Framework, launched in 2021, aims to improve the lives of those affected by rare conditions through enhanced diagnosis, treatment, and support.
Patient advocacy groups play a crucial role in raising awareness and supporting those affected by rare diseases. Events like Rare Disease Day, observed annually on 29 February (or 28 February in non-leap years), bring global attention to these conditions. Personal stories of individuals living with rare diseases, such as cystic fibrosis, Huntington's disease, and Duchenne muscular dystrophy, highlight the daily challenges and triumphs experienced by patients and their families.
The history of rare disease research dates back to the early 20th century, with significant milestones including the discovery of alkaptonuria by Sir Archibald Garrod in 1902. The Orphan Drug Act of 1983 in the United States and similar legislation in other countries have since incentivised pharmaceutical companies to develop treatments for rare conditions, leading to a surge in orphan drug approvals over the past few decades.
Staying informed about rare diseases is crucial for patients, healthcare professionals, and policymakers alike. Our NewsNow feed provides comprehensive, up-to-date coverage from reliable sources, ensuring you have access to the latest breakthroughs, policy changes, and patient support initiatives in the field of rare diseases.