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New drug offers potential cure for ultra rare inherited condition
A teenage patient who helped discover a rare condition is the first to benefit from a new treatment.
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bbc.com
New drug offers potential cure for ultra rare inherited condition
A teenage patient who helped discover a rare condition is the first to benefit from a new treatment.
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telegraph.co.uk
Teenager is first person in Europe to get wonder drug for rare genetic disorder
Mary Catchpole, 19, receives ‘life-changing’ treatment for immune condition after NHS approval