Are we paying too much for too little? @VPrasadMDMPH discusses the use of nusinersen for spinal muscular atrophy #HotTopicsNeo2020 #neoEBM 
Monthly costs for orphan drug prices for rare conditions
The cost for nusinersen, an intrathecal therapy for SMA, costs approximately $125,000 per dose
Here is a commentary by @VPrasadMDMPH in @JAMAPediatrics on this topic jamanetwork.com/journals/jamap…
Dr. Prasad notes switching of primary end-point in ENDEAR trial of nusinersen (nejm.org/doi/full/10.10…) from mortality/ventilation to Hammersmith neurologic exam score when pursuing FDA approval
Some of the ethical challenges regarding nusinersen treatment for spinal muscular atrophy by @BurgartBioethix jamanetwork.com/journals/jamap…
Some general lessons by @VPrasadMDMPH regarding the challenges in approaches to drug approval for rare diseases
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