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pro vyhledávání: '"Maria E, Herva"'
Autor:
Blanca, Torroba, Natsuko, Macabuag, Elisabeth M, Haisma, Amy, O'Neill, Maria E, Herva, Roxana S, Redis, Michael V, Templin, Lauren E, Black, David F, Fischer
Publikováno v:
Expert Opinion on Drug Discovery. 18:181-192
Spinal Muscular Atrophy (SMA), the second most prevalent autosomal genetic disease affecting infants, is caused by the lack ofThis manuscript will cover two of the three mechanistically distinct available treatment options for SMA, both targeting the