Browsing Dahili Tıp Bilimleri Bölümü Makale Koleksiyonu by Author "Finkel, Richard S."

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    • Nusinersen Initiated In Infants During The Presymptomatic Stage Of Spinal Muscular Atrophy: Interim Efficacy And Safety Results From The Phase 2 Nurture Study 

      De Vivo, Darryl C.; Bertini, Enrico; Swoboda, Kathryn J.; Hwu, Wuh-Liang; Crawford, Thomas O.; Finkel, Richard S.; Kirschner, Janbernd; Kuntz, Nancy L.; Parsons, Julie A.; Ryan, Monique M.; Butterfield, Russell J.; Topaloglu, Haluk; Ben-Omran, Tawfeg; Sansone, Valeria A.; Jong, Yuh-Jyh; Shu, Francy; Staropoli, John F.; Kerr, Douglas; Sandrock, Alfred W.; Stebbins, Christopher; Petrillo, Marco; Braley, Gabriel; Johnson, Kristina; Foster, Richard; Gheuens, Sarah; Bhan, Ishir; Reyna, Sandra P.; Fradette, Stephanie; Farwell, Wildon (2019)
      • NURTURE is an ongoing study of nusinersen started in a presymptomatic stage of SMA. • All infants were ≥25 months old, and alive without permanent ...