The knowledge we are gaining at n-Lorem has even more value than benefiting nano-rare patients and their families. These insights will enable scientists to discover new therapeutic targets for both common and rare diseases, fundamentally changing the way we approach health and disease.

On This Episode We Discuss:

• Two most common words in medicine: Health and Disease
• The Scientific Method
• The Importance of Single Variables
• Orthogonal Thinking
• We Are at a Unique Moment in Medical History
• Genomics
• Advances in Omics
• Biological Networks and AI
• Facile Collection, Maintenance, Growth and Differentiation in the Lab
• Antisense Technology

Paul Compton and his wife Andrea chose to start a family through adoption. Unknown to anyone, one of their adopted boys, Taeson, was the inheritor of DRPLA, a progressive brain disorder caused by a mutation in the ATN1 gene. Determined to find a treatment and advocate for their son and others with the disease, the Comptons established CureDRPLA. In early 2024, Taeson received his initial treatment with an ASO discovered and developed by n-Lorem. Paul discusses his family’s journey and shares his observations of Taeson's progress since beginning treatment.

In This Episode We Discuss:

3:05 – Paul's origins in business and finance

4:32 - Investment banking is different than traditional banking

7:27 – Opposites attract – Meeting his wife, Andrea, and starting a family through adoption

10:06 – Paul’s son, Tayson, has a progressive brain disorder known as DRPLA, which is caused by a mutation in the ATN1 gene; Discovering the idea of n-Lorem

16:03 – Tayson’s journey to a diagnosis

23:00 – The deterioration of his abilities over the years

25:00 – The progress Paul has observed in his son since initial treatment

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Mutations affecting ion channels are the most common cause of 'applications for treatment' submitted to n-Lorem. These channels regulate the passage of essential electrically charged ions, like sodium, potassium, calcium, and chloride, into cells. Alas, ion channels pose a significant challenge in the ASO discovery process, as there is little room for error due to the need for highly allele-selective ASOs to achieve success. Let’s 'dive into the channel' and explore the complexities of treating patients with ion channel mutations.

Recently a report on one of our patients with an ion channel mutation was published in Endpoints News highlighting the power of our technology for these disorders. Check it out by clicking the link below.

Endpoints News (endpts.com) A teenager faced constant seizures. Could a drug developed just for him stop them?

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On This Episode We Discuss:

• Defining Ion Channels and Ions
• Health, Homeostasis, and Biological Buffering
• Multiple forms of the same gene
• Multiple Isoforms of Gene Product from the same gene
• Network redundancy
• Ion Channels are different