Results 1 - 1 of 1 for Nusinersen
  1. Gene therapies are a promising path to treating rare diseases | NIH MedlinePlus Magazine 
    ... Genetic therapies to improve movement A medication called nusinersen was one of the first genetic therapies approved ... those problems in children and adults with SMA. Nusinersen targets another gene called SMN2 that also makes ...
    https://magazine.medlineplus.gov/...g-path-to-treating-rare-diseases - MedlinePlus Magazine