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The median life expectancy for infants with SMA presenting before 6 months of age is less than 2 years without respiratory support. To date, there is no cure for SMA. In June 2017, nusinersen was approved in Canada as the first disease\u2010modifying drug for SMA because of its demonstrated benefits on motor function and survival in clinical trials. However, with a price tag of almost 1 million dollars for the first year of therapy, careful clinical, treatment\u2010based and ethical consideration of the principles of (i) best interests; (ii) universality; (iii) portability; (iv) public administration; (v) accessibility; and (vi) comprehensiveness are important guideposts to ensure transparent and equitable allocation of health\u2010care resources for nusinersen and all other future orphan drugs.<\/jats:p>","DOI":"10.1111\/jpc.14226","type":"journal-article","created":{"date-parts":[[2018,9,24]],"date-time":"2018-09-24T01:32:47Z","timestamp":1537752767000},"page":"18-24","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Putting our best foot forward: Clinical, treatment\u2010based and ethical considerations of nusinersen therapy in Canada for spinal muscular atrophy"],"prefix":"10.1111","volume":"55","author":[{"given":"Sonya","family":"Vukovic","sequence":"first","affiliation":[{"name":"Faculty of Medicine University of Toronto  Toronto Ontario Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laura","family":"McAdam","sequence":"additional","affiliation":[{"name":"Division of Developmental Pediatrics University of Toronto  Toronto Ontario Canada"},{"name":"Bloorview Research Institute The Hospital for Sick Children  Toronto Ontario Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Randi","family":"Zlotnik\u2010Shaul","sequence":"additional","affiliation":[{"name":"Department of Bioethics The Hospital for Sick Children  Toronto Ontario Canada"},{"name":"Department of Pediatrics University of Toronto  Toronto Ontario Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Reshma","family":"Amin","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Toronto  Toronto Ontario Canada"},{"name":"Division of Respiratory Medicine The Hospital for Sick Children  Toronto Ontario Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2018,9,23]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(80)90847-8"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/8.7.1177"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/B978-0-12-803685-3.00025-2"},{"key":"e_1_2_6_5_1","unstructured":"Cure Spinal Muscular Atrophy.Cure SMA Breathing.2018. 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