Patient Empowerment Program: A Rare Disease Podcast
n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen)
85 episodes
1 month ago
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. Crooke, who is the Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Andrew Serrano, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org.
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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. Crooke, who is the Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Andrew Serrano, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org.
Patient Empowerment Program: A Rare Disease Podcast
49 minutes 30 seconds
8 months ago
Rosie’s Story: A Nano-rare Treatment Pioneer
Rosie was the first of three HNRNPH2 patients to receive treatment with an n-Lorem ASO, helping to pave the way for others. Her parents, Nicole and Bobby, share their daughter's journey and how they have adapted as a family to meet her unique needs. As a pediatrician, Nicole understands the complexities of medical care, yet she acknowledges that even she doesn’t always have the answers when it comes to raising a child with a nano-rare disease—a reality that resonates with many nano-rare families.On this episode we discuss:
2:00 Bobby left his job to help care for Rosie full-time
7:36 Nicole is a pediatrician and it’s difficult for her to not regularly attend Rosie’s appointments
11:16 Rosie’s diagnosis of HNRNPH2
16:00 Even as a pediatrician, Nicole finds nano-rare diseases complex and difficult to navigate
19:29 What is HNRNPH2?
26:40 Coming to terms with the diagnosis
29:35 Weighing the potential risk/benefit and making the decision that Rosie will be the first HNRNPH2 patient treated with an experimental medicine
37:05 How Rosie is doing after three ASO treatment doses
40:35 Potential for a placebo effect and allowing multiple observers to notice changes
Donate to n-Lorem: https://www.nlorem.org/donate/ This episode is brought to you by Hongene Biotech: https://www.hongene.com/
Patient Empowerment Program: A Rare Disease Podcast
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. Crooke, who is the Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Andrew Serrano, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org.
