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204 results
  • GeneReviews®: Spinal Muscular Atrophy [BOOK]
    University of Washington, Seattle: Seattle (WA)Adam MP, Ardinger HH, … Amemiya APrior TW, Finanger E, … Leach MEBOOK
  • Spinal muscular atrophy (SMA) is characterized by muscle weakness and atrophy resulting from progressive degeneration and irreversible loss of the anterior horn cells in the spinal cord (i.e., lower motor neurons) and the brain stem nuclei. The onset of weakness ranges from before birth to adulthood. The weakness is symmetric, proximal > distal, and progressive. Before the genetic basis of SMA wa…
  • Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives. [Journal Article]
    Ther Clin Risk Manag 2019; 15:1153-1161Dangouloff T, Servais L
  • Recent advances in the treatment of spinal muscular atrophy (SMA) have dramatically altered prognosis. Rather than a rapidly lethal disease, SMA type 1, the most severe form with the earliest onset of SMA, has become a disease in which long-term event-free survival with the acquisition of important motor milestones is likely. Prognosis for patients with SMA type 2 has shifted from slow and progre…
  • One Year of Newborn Screening for SMA - Results of a German Pilot Project. [Journal Article]
    J Neuromuscul Dis 2019; 6(4):503-515Vill K, Kölbel H, … Müller-Felber W
  • CONCLUSIONS: Newborn screening, resulting in presymptomatic treatment, improves outcome in children with genetically proven SMA. Newborn screening for SMA should be introduced in all countries where therapy is available. An immediate therapy in cases with 4 SMN2 copies should be considered.
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