When patients face the diagnosis of a rare disease, they face a costly, uphill battle to develop a treatment for their condition… that is, if they ever do.  

Dr. Stanley Crooke is aiming to change that. As the CEO of n-Lorem Foundation, his mission is to provide personalized treatments to ultra-rare disease patients for free as long as they live, no questions asked. Thanks to his humility, coupled with the generosity of other individuals and companies, ultra-rare disease patients have a renewed hope to get the treatments others may not be able to provide.  

Join Dr. Crooke as he reflects on his humble beginnings at Ionis, the approach n-Lorem takes to developing drugs for ultra-rare diseases, and what the future holds for research in this area.

Show Notes 

• n-lorem Foundation 
• Charles River - Rare Disease 
• Vital Science S4, E01 Project ALS: Fueled by Love 
• 2021 Rare Disease Trailblazers 
• BioSpace: n-Lorem Foundation Preps First Doses to Treat Ultra-Rate Disease Patients for Free 
• Ionis Pharmaceuticals 
• Nature Medicine: Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis