Two years ago, Beatrice Cieslik was diagnosed with CLN2 type Batten disease - a rare degenerative condition which causes ...
Parents of children with a rare neurodegenerative disease say they are in disbelief they could within months lose access to ...
The news was welcomed by the nine-year-old’s family, who saying stopping the medication would hasten her death.
The B.C. government has decided to fund the medication for Charleigh until a review of the decision to end funding is ...
The benefits of Biomarin’s Brineura for a very rare inherited condition do not justify its price of more than £520,000 per year, NICE has said in first draft guidance. NICE had been assessing ...
She is currently treated with the drug Brineura, allowing her to attend school and live "a better quality of life". NHS access to the drug is set to end in May, but the National Institute for ...
Flory, six, is thriving on her treatment of Brineura, her mum says Parents of children with a rare neurodegenerative disease say they are in disbelief they could within months lose access to the ...
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