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Patient Empowerment Program: A Rare Disease PodcastAuthor: n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen)
Join the nano-rare disease community! Interviews features leading physicians, scientists, biotech experts, and patient advocates. Lessons teach core concepts about drugs. Our host Dr. Crooke has led the creation of antisense technology and his foundation, n-Lorem, is using this powerful technology to discover, develop, and provide personalized experimental antisense oligonucleotide medicines to nano-rare patients for free, for life. n-Lorem is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients (less than 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. n-Lorem was founded by Dr. Stan Crooke, who founded IONIS Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. The podcast is produced by n-Lorem Foundation and hosted by Dr. Stanley T. Crroke, who is the Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Kim Butler, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org. Language: en Genres: Education, Life Sciences, Science Contact email: Get it Feed URL: Get it iTunes ID: Get it Trailer: |
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Your Questions, Answered: 2026 Q&A
Episode 88
Wednesday, 14 January, 2026
Your questions, answered. n-Lorem CEO, Stan Crooke, answers questions directly from the nano-rare community in this special Q&A episode. How often is Drug safety monitored? When is an existing ASO made available to others? Not capping accepted applications and more. Do you have additional questions? Email podcast@nlorem.org. On This Episode We Discuss: 5:40 - What is the frequency range of intrathecal and/or LP administrations? Do the treatments not typically cross the blood brain barrier? 7:06 - How do you administer the ASO to animals - is it according to how you plan to administer to the patient? 7:36 - How do you evaluate the potential on-target toxicity if the nonclinical species does not contain the targeted sequence? 9:35 - How often is safety monitored? Is it only monitored quarterly at the Data/Drug Safety Monitoring Board (DSMB) meetings? 11:03 - How long after an ASO is developed for one child, is it offered to other children? Of course, we know that they will have to submit an application. Does the ASO become potentially available for other children after the first child's 2-year trial is over? Or is it potentially available after just a few doses to confirm safety and efficacy? 14:18 - Are there plans to commercialize drugs for the nano-rare? 16:27 - What proportion of institutions elect to use external / centralized institutional review boards (IRBs)? 18:21 - Will there be a cap of accepted applicants from n-Lorem? The demand will only grow and there’s a fear for some families that there’s a chance of n-Lorem capping the number? 19:44 – Once we have our neurologist team working on getting our child accepted, what else do we need to do? What I guess is our next step? Is there anything we should do in the meantime? 20:31 – What should a parent expect once their application has been accepted to n-Lorem? 21:19 – There are widely varying timelines regarding this process. What as a parent should I expect regarding timing? 22:30 – There are companies and academic scientists that claim to provide ASOs for a fee. What do you think about these providers? 23:40 – Insights: What is n-Lorem learning that will be broadly applicable to the scientific community; Dreaming bigger and hoping for more
