When Luke Rosen's daughter was diagnosed with KIF1A—a rare, progressive neurological disorder—he didn't wait for answers. He and his wife built a community, launched a nonprofit, and became a driving force in rare disease research. In this episode, Luke shares how patient-led science accelerates treatments and reshapes what’s possible for families like his.
Show Notes
Sounds of Science - N=1 Episode
Charles River | Rare Disease
Charles River | Rare Disease for Drug Discovery
Charles River | ASO Screening Services
Personalized ASO Provides Improvement for a Girl with KAND< an Ultra-rare Disease