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The Journey of Susannah’s Personalized ASO
Manage episode 480860705 series 2304454
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
95 episodes
Manage episode 480860705 series 2304454
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
95 episodes
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