Conclusion Patients with SMA1 treated with nusinersen initially spend a considerable proportion of their early life in hospital. Parents should be counselled accordingly. These data suggest that for every 10 children started on nusinersen an extra HDU bed is required. This has a significant cost ...
It is well established that starting treatment as early as possible, preferably before symptom onset, is key for achieving best outcomes and reducing the overall SMA treatment cost.The type of treatments a patient may need depends on the severity of symptoms and should be determined together with...
It is well established that starting treatment as early as possible, preferably before symptom onset, is key for achieving best outcomes and reducing the overall SMA treatment cost.The type of treatments a patient may need depends on the severity of symptoms and should be determined together with...
let you know the rare disease SMA 中⽂名：脊髓型肌萎缩症 英⽂名：spinal muscular atrophy(简称SMA)别名：Werdnig-Hoffman病；Dubowitz病 Kugelberg-Welander病 突变基因：SMN1 疾病科室：神经内科 English name: spinal muscular atrophy (SMA for short)Aliases: Werdnig-Hoffman disease; Dubowitz disease; ...
Zolgensma costs $2.1 million for the one-time-only treatment. The reason Zolgensma is so expensive is because that is the price Novartis has decided it is worth because it “dramatically transforms the lives of families affected by this devastating disease” and the claimed cost of bringing ...
"This important step by the FDA brings us ever closer to delivering Zolgensma to patients with SMA Type 1. Babies affected by this rare disease are currently faced with debilitating disease progression and lifelong invasive chronic treatment. As a one-time infusion that addresses the genetic root...
I think one of the things that’s very challenging about this scenario, before I get to just talking about cost, is assessment of value. We don’t know what the longevity and durability of the treatment are. People usually think about longevity in terms of the patient. But here, as we...
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In 2021, NICE recommended that Zolgensma be covered by the NHS given its potential to substantially prolong and improve the lives of babies with SMA, despite its high cost. But that recommendation only covered infants as old as 1 year who had been diagnosed with SMA type 1 and who did no...