“Building the plane while flying”: how real-world evidence is accelerating treatment options for people with spinal muscular atrophy
In this article, Peter Persson and Ben Tichler (both Biogen) discuss the role of real-world evidence in rare diseases, specifically spinal muscular atrophy, and how real-world data can be used to accelerate treatment options for people with rare diseases. “It’s like clinical trials provided the fuselage and wings, and real-world…