Although CLN2 disease is an ultra-rare disease based on prevalence (about one in two million), its incidence (about one in 200,000) suggests a higher number of treatable patients. Payers in France ...

Results showed a strong but non-linear relationship between disease prevalence and AATC for drugs recommended by NICE – with drugs for the treatment of ultra-rare diseases achieving a higher AATC.

February 28, marks Rare Disease Day, a global movement working towards equity in health care and access to diagnosis for people living with a rare disease. Although definitions vary, a disease is ...

A study led by UCL found that individuals with rare diseases were five times more likely to die from Covid-19 than the ...

An England-wide study of over 58 million people has identified eight rare diseases that carry significantly increased risks for COVID-19-related mortality in fully vaccinated individuals. This ...

An England-wide study of 331 rare diseases in over 58 million people co-led by UCL researchers has identified eight rare ...

Researchers in the UK are calling for better inclusion of rare diseases in public health strategies, including pandemic planning, vaccination policies, and National Health Service (NHS) service ...

Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance ...

Alkaptonuria is a rare genetic disorder that causes black urine due to homogentisic acid buildup, leading to joint pain, ...