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.
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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.
Be Relentless In Seeking Help
- Luke Rosen recommends learning from diseases already treated with ASOs (like SMA) and then persistently contacting experts and companies.
- He credits relentless outreach—calling Stan Crooke repeatedly—for getting Susanna into a treatment program.
Two-Year Wait And The Anesthesia Fear
- It took roughly two years from acceptance into n-Lorem to Susanna's first ASO dose, and Luke recalls the anxiety of her anesthesia before dosing.
- He forced a reassuring smile as she fell asleep, hoping the treatment would work and not be their last peaceful moment.