The spinal muscular atrophy (SMA) pipeline measures the progress of our research in two key ways. First, by tracking each individual program all the way to U.S. Food and Drug Administration (FDA) approval. Second, by tracking how these programs are spread among the different therapeutic approaches.
Progress of Individual Drug Programs
Cure SMA’s research model funds drug programs at all stages of development. Our basic research program studies the biology and causes of SMA, often revealing new and more effective ways of making drugs. These basic research ideas are then converted into practical drug candidates through drug discovery. Finally, those drug candidates move through the clinical trial process. Our drug pipeline monitors each individual program as it moves through these stages.
Approved Therapies for SMA
Several therapies have been approved for SMA: Evrysdi, marketed by Genentech/Roche; Spinraza, marketed by Biogen; and Zolgensma, marketed by Novartis Gene Therapies. The drug pipeline also tracks the implementation of these and other therapies as they are approved.