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    1. Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular (...)

      by Montes, Jacqueline; Bennett, C. Frank; De Vivo, Darryl C.; Norris, Daniel A.; Bishop, Kathie M.; Chiriboga, Claudia A.; Swoboda, Kathryn J.; Darras, Basil T. and Iannaccone, Susan T.

      Neurology, Volume 86, Issue 10, pp. 890 - 897

      Journal Article : Full Text Online

    2. Spinal muscular atrophy — insights and challenges in the treatment era

      by Muntoni, Francesco; Pera, Maria Carmela; Scoto, Mariacristina; Finkel, Richard and Mercuri, Eugenio

      Nature Reviews. Neurology, Volume 16, Issue 12, pp. 706 - 715

      Journal Article : Full Text Online

    3. Prevalence, incidence and carrier frequency of 5q–linked spinal muscular atrophy – a (...)

      by Aartsma-Rus, Annemieke; Verhaart, Ingrid E. C.; Cook, Suzanne F.; Robertson, Agata; Lochmüller, Hanns; Wilson, Ian J.; Cameron, Shona and Jones, Cynthia C.

      Orphanet Journal Of Rare Diseases, Volume 12, Issue 1, p. 124

      Journal Article : Full Text Online

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    1. Spinal muscular atrophy : disease mechanisms and therapy

      edited by Charlotte J. Sumner, Sergey Paushkin, Chien-Ping Ko.

      Book : View Online

    2. Spinal muscular atrophy fact sheet.

      Government publication, Web page or site : View Online

    3. New Hope for Spinal Muscular Atrophy

      Ivanhoe Broadcast News.

      Video : View Online

    See all 93 Books & Media results

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