Medical research

Low enrollment stymies completion of rare disease clinical trials

Rare diseases are anything but rare when taken together. In the United States alone, they affect nearly 30 million people. Yet clinical trials for rare diseases are frequently incomplete or go unpublished in the medical literature, ...

Medications

Scientists suggest new solution to the rare-disease problem

Thousands of rare diseases cumulatively affect millions of people across the globe, yet because each case is so rare doctors struggle to accurately diagnose and effectively treat individual patients. Every time a patient ...

Medical research

Possible new treatment strategy against progeria

Progeria is a very rare disease that affects about one in 18 million children and results in premature aging and death in adolescence from complications of cardiovascular disease. In a study on mice and human cells, researchers ...

Medical research

Pulmonary arterial hypertension targeted for new treatment

Scientists at the University of Sheffield, working in collaboration with drug and vaccine developer Kymab Ltd, Cambridge, have identified a novel antibody that has the potential to become a new treatment for pulmonary arterial ...

Genetics

Rare diseases: Over 300 million patients affected worldwide

Rare diseases represent a global problem. Until now, the lack of data made it difficult to estimate their prevalence. Created and coordinated by Inserm, the Orphanet database contains the largest amount of epidemiological ...

Diseases, Conditions, Syndromes

Cause of paralyzing illness in kids remains elusive

(HealthDay)—There is still no clear cause for a mysterious paralytic condition that has been striking U.S. children over the past five years, government health officials report.

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