Sounds of Science

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 
SCA3: A Family Affair 
Susannah's Superhero Story 

What is Sounds of Science?

Sounds of Science is a monthly podcast about beginnings: how a molecule becomes a drug, how a rodent elucidates a disease pathway, how a horseshoe crab morphs into an infection fighter. The podcast is produced by Eureka, the scientific blog of Charles River, a contract research organization for drug discovery and development. Tune in and begin the journey.