Drug trial offers hope for genetic disorder first discovered in Cumbria
by Jane Kirby
Mar 21, 2024
3 minutes
Scientists are hoping to repurpose an existing drug to treat an ultra-rare genetic disorder that has plagued families living in the North of England.
Neuroferritinopathy is a progressive and incurable brain condition, whose symptoms including loss of speech and swallowing.
It is thought to affect about 100 people worldwide.
The condition was discovered more than 20 years ago when a British scientist began studying a family in Cumbria who thought they had an inherited
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