The European Commission approves the Western hemisphere’s first gene therapy, aimed at correcting a lipid-processing disorder.
The European Commission approves the Western hemisphere’s first gene therapy, aimed at correcting a lipid-processing disorder.
People living on islands in the Norwegian Sea suffer from an unusually high rate of certain genetic diseases and health issues, making the population ripe for research.
New congressional proposals would allow drug makers to bypass traditional clinical trial requirements for drugs that address rare diseases.
A Gulf of California fisherman brings home a baby dusky shark with only one eye.
With mounting interest from biotechs, Big Pharma, and the federal government, research on rare diseases is burgeoning.
Two research teams studying a rare genetic disorder discover independently that it's caused by genes that are crucial to DNA replication.
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