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U.K. Infants With SMA Type 1 Promised Nusinersen Treatment


The Great Ormond Street Hospital in London, England has teamed up with pharmaceutical giant, Biogen, to offer infants with spinal muscular atrophy (SMA) type 1 treatment using the drug Nusinersen (Spinraza) under their Expanded Access Program until the drug becomes licensed in the U.K.

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One of the lucky recipients of the new wonder drug is 1-year-old Sophia Hart from Essex, England. Hart was diagnosed with SMA type 1 just before her first birthday and her parents feared the worst for their little girl.

According to a report in the East London and West Essex Guardian, Sophia’s family is already seeing the effects of the drug. She is now able to sit unaided and hold a cup — two important milestones her doctors and parents didn’t think she would ever reach.

Nusinersen is not yet available via the National Health Service in the U.K., and won’t be until the National Institute for Health and Clinical Excellence (NICE) produces a report on the effectiveness of the product.

MORE: From idea to injection: how Spinraza came to be 

SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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