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.
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.
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