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  • nusinersen
  • The objective of this study was to evaluate the cost-effectiveness of nusinersen for the treatment of patients with infantile-onset SMA and later-onset SMA in Sweden. (rtihs.org)
  • For a time horizon of 40 years in the infantile-onset model and 80 years in the later-onset model, treatment with nusinersen resulted in 3.86 and 9.54 patient incremental quality-adjusted life-years (QALYs) and 0.02 and 2.39 caregiver incremental QALYs and an incremental cost of 21.8 and 38.1 million SEK (Swedish krona), respectively. (rtihs.org)
  • Treatment with nusinersen results in overall survival and QALYs benefits but with incremental costs above 21 million SEK (mainly associated with maintenance treatment with nusinersen over a patient's lifespan), which translate into incremental cost-effectiveness ratios (including caregiver QALYs) of 5.6 million SEK and 3.2 million SEK per QALY gained in the infantile-onset model and later-onset model, respectively. (rtihs.org)
  • The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). (clinicaltrials.gov)
  • The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA and to examine the cerebral spinal fluid (CSF) and plasma Pharmacokinetics (PK) of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA. (clinicaltrials.gov)
  • The U.S. Food and Drug Administration (FDA) approved nusinersen (Spinraza) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients (Biogen, 2016). (aetna.com)
  • The data package included the interim analysis of ENDEAR, a Phase 3 controlled study evaluating nusinersen in infantile-onset, as well as open-label data in pre-symptomatic and symptomatic patients with, or likely to develop, Types 1, 2 and 3 SMA. (aetna.com)
  • hypotonic
  • The infant with botulism becomes progressively weak, hypotonic and hyporeflexic, showing bulbar and spinal nerve abnormalities. (aafp.org)
  • Physical
  • Morbidity associated with these conditions often can be minimized with spinal surgery, as well as with aggressive physical therapy. (medscape.com)
  • Childhood
  • Soon after, Professor Johann Hoffmann from Heidelberg University presented a paper describing a syndrome of progressive atrophy, weakness, and death during the early childhood period of siblings with genetically normal parents. (medscape.com)
  • Study
  • This qualitative study provides an in-depth account of the range and nature of costs incurred by families caring for a child with spinal muscular atrophy (SMA) and has identified costs not previously described or under-reported by quantitative studies. (bmj.com)
  • particularly
  • Anaerobic exercise may be substituted for high-intensity muscular activity, particularly when blood flow is reduced and oxygen availability is limited. (medscape.com)