New Data presented at the Muscular Dystrophy Association’s clinical conference show benefit in motor function for infants, teens and young adults treated with Spinraza™ (nusinersen).
The NURTURE study results reported that all study participants (25 infants) were alive and experienced improved motor function at the time of reporting, compared to a decline seen in SMA natural history.
One-hundred percent of NURTURE participants achieved the age-expected World Health Organisation motor milestone of sitting without support, a development never seen with SMA Type 1.
Case series conducted on Spinraza-treated teens and young adults with SMA Type 2 and 3 (17 to 19 years old upon their last visit) demonstrated stable or improved motor function and improved quality of life.
Spinraza™ is the first treatment for SMA. It was approved for the treatment of all SMA Types by the European Commission on 1st June 2017, after recommendation by the European Medicines Agency.