Eric Hoffman gave a great talk on how the community around Duchenes Muscular Dystrophy has driven research into treatments. This talk was very inspiring and touching, especially given the context of the first treatments for DMD recently being approved by the FDA.
Eric is the founder and CEO of Reveragen, which is developing Vamorolone, a replacement for conventional steroids. It is targeted at DMD but is also a potential treatment for SJIA. The drug was recently fast-tracked by the FDA.