World Rare Disease Day, an annual international effort to create awareness for rare diseases, will take place on February 28th. This year, the theme is on how research brings hope to people living with rare diseases. This seemed like an opportune time to talk to Kenneth Hobby, President of CureSMA, about his organization’s efforts to drive research for spinal muscular atrophy, the most common genetic cause of death for infants. We spoke to Hobby about CureSMA’s strategy for research, the role the group played in helping to make possible the first approved therapy for the disease at the end of last year, and what other rare disease organizations can learn from its experience.
RARECast is a weekly series by Daniel S. Levine. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013, which produces The Bio Report and RARECast podcasts. Read his full bio here.