Tailor-made drug shows success in Batten disease
The process could pave the way for more rapid delivery of individualised treatments.
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Tag: Batten disease
A&E attendances twice as high in deprived areas
Attendances for the 20% of the population living in the most deprived areas also account for 27% of all A&E attendances.
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NICE deems Batten disease therapy too costly for NHS use
The National Institute for Health and Care Excellence has issued draft guidelines concluding that Biomarin’s Brineura is not a cost-effective treatment option for patients with the ultra rare disorder Batten disease.
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US clears first therapy for Batten disease
US regulators have approved BioMarin Pharmaceutical’s Brineura as the first treatment for a rare inherited form of Batten disease.
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