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商品編號： 9-621-112 出版日期： 2021/05/19 作者姓名： Chandra, Amitabh;Lee-Rey, Spencer;Marra, Caroline 商品類別： Other 商品規格： 19p 再版日期： 地域： United States 產業： Healthcare sector;Public administration 個案年度： -
商品敘述：
This case explores incentives for rare disease drug development by chronicling the role of the Spinal Muscular Atrophy (SMA) Foundation in forming strategic partnerships with the scientific research community and pharmaceutical developers to transform the trajectory of innovation for the disease. In the 17 years since its inception, the Spinal Muscular Atrophy (SMA) Foundation has helped transform SMA from an underfunded and poorly researched disease with no available treatments to one with three novel drugs approved by the Food and Drug Administration (FDA). While these therapies greatly improved the quality of life for many newly diagnosed patients, there was still a substantial portion of existing patients with unmet medical needs. Though many in the medical community, and even at the FDA, felt that SMA had been all but cured, the Foundation could not accept that their work was done until every patient had a treatment option.
涵蓋領域：
Business and society;Business management;Health and wellness;Innovation;Inventions;Strategy
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