Squawk Pod The Path with Becky Quick: Finding Hope 1/30/26
Jan 30, 2026
Dr. Stanley Crooke, founder of n-Lorem and pioneer of antisense oligonucleotide therapy, and Luke Rosen, parent-advocate who founded KIF1A.org after his daughter’s diagnosis. They discuss ultra-rare KIF1A disease, how personalized ASO treatments are developed and delivered, the emotional and logistical journey of seeking care, and barriers families face accessing research and therapies.
AI Snips
Chapters
Transcript
Episode notes
Bath Moment That Changed Everything
- Luke Rosen describes noticing Susanna couldn't kick in the bath and rushing her to urgent care at age two and a half.
- That moment started their search for diagnosis and the creation of KIF1A.org to find treatments.
KIF1A's Toxic Gain Of Function
- KIF1A is a vital molecular motor protein whose toxic gain-of-function mutation progressively kills neurons.
- That biological mechanism explained Susanna's ataxia, seizures, and likely future wheelchair dependence.
The Nanorare Gap In Drug Development
- Sequencing reveals a spectrum of nanorare pathogenic mutations affecting very few humans worldwide.
- These patients are isolated and overlooked by commercial drug development despite having devastating diseases.