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Spinal Muscular Atrophy News

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The cost burden of standard of care for patients with spinal muscular atrophy (SMA) is substantial, and at current prices approved disease-modifying drugs for the condition have high cost-effectiveness ratios when delivered after symptom onset. Researchers published findings from their systematic…
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Illness costs resulting from spinal muscular atrophy (SMA) vary widely across disease phenotypes and geographical settings, according to a systematic literature review published online ahead of print in the journal Applied Health Economics and Health Policy.  “There are several phenotypes of SMA…
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The gene therapy drug onasemnogene abeparvovec for spinal muscular atrophy appears efficacious and well tolerated in infants older than 7 months, according to a study published online ahead of print in Pediatric Neurology.  “Onasemnogene abeparvovec was recently approved for the treatment of…
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Improvements in motor function and breathing function are among treatment attributes most preferred by patients with spinal muscular atrophy (SMA) and their caregivers, according to a study published online in the Orphanet Journal of Rare Diseases.  “This study is the first-ever discrete choice…
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For nusinersen to be cost-effective without newborn screening for infantile-onset spinal muscular atrophy, the cost of the drug would need to be reduced substantially, according to a study published in The Journal of Pediatrics.  “Universal newborn screening for spinal muscular atrophy provides…
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Inpatient and outpatient costs directly associated with spinal muscular atrophy (SMA) averaged more than $100,000 annually per patient from 2012 to 2016, according to a study published in the Journal of Market Access & Health Policy. “Direct costs of SMA are tremendous, often more than 50-fold…
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Following the recent FDA approval of Spinraza (nusinersen; Biogen), the manufacturer has announced the treatment will cost patients with spinal muscular atrophy $750,000 for the first year of treatment, with additional annual treatment costs of  $375,000. Spinraza treats a rare spinal disorder in…
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