Q&A #2 with Patient Families

February 7, 2024


Stan sits down with patient families to have a conversation and together address additional questions from the 2023 Nano-rare Patient Colloquium.

Charissa Lipman joins n-Lorem founder and CEO, and host of the Patient Empowerment Program Podcast, Dr. Stan Crooke, in this question-and-answer episode to discuss additional questions asked during the 2023 Nano-rare Patient Colloquium. Your diverse array of questions reflects not just an interest in the present, but a keen anticipation of what lies ahead as personalized medicine continues to advance and evolve. Together, Stan and Charissa address your curiosities about n-Lorem, ASOs, and nano-rare. Charissa attended the inaugural Colloquium and brings the perspective of a patient family member, discussing her experiences and takeaways from the meeting. She is the mother of Ryker, a nano-rare n-Lorem patient with a CACNA1A genetic mutation. 

Do you have a question you want to ask Stan Crooke? Email [email protected] for a chance to be featured in a future episode. 

For general questions, email [email protected].

Question Bank:

  1. 08:25 As you are successful in discovering and developing individualized ASO for nano-rare patients, do you envision creating a library of ASOs that would be available to patients across the world?
  2. 10:29 For disease organization/patient advocacy groups that have several patients with the same mutation, should they apply for treatment as a group or separately?
  3. 13:16 Would an ASO developed for one patient work for another patient with a mutation in the same gene?
  4. 14:29 What’s a SNP?
  5. 16:19 As a nano-rare family member, I have never been so inspired in an opening address as what was provided at the 2023 colloquium by Stan Crooke. Given the mission of n-Lorem, I don’t understand how any researching neurologist (or any researcher in the space) would not want to be at the absolute forefront of what n-Lorem is doing. Why do you think there is such an obstacle to being a part of an organization that has for the first time an opportunity to move the needle in such a meaningful way?
  6. 20:16 How would you describe the relationship between n-Lorem and the research physician, and what should patients expect from each side?
  7. 23:44 What do you mean by an optimized ASO?
  8. 24:59 Would you expect to see better results from ASO treatment in patients who are younger versus older?
  9. 28:10 The FDA has certain designations for program review (Fast Track, Orphan, etc,), is there anything like that for nano-rare patients?
  10. 29:35 I realize that there was significant work done in 2023 to streamline n-Lorem’s operations. Which processes in the workflow do you believe can still be optimized to help streamline patient programs?
  11. 31:35 Has there been a change in the amount of time it takes to process a patient and develop and ASO since the Foundation started?
  12. 32:50 Do you reach out to patients which presumably have ‘ASO-able’ genetic mutations?
  13. 34:12 Do you have any activities or ongoing projects that would help educate and train physicians who might be interested in participating with n-Lorem?
  14. 36:05 Are there any new updates regarding the 2024 Nano-rare Patient Colloquium?
  15. 41:36 Say a patient is denied from n-Lorem because at the time of their application submission, the Access to Treatment Committee declares that the program is not amendable to the current technology and/or ASO strategies, but new data/technology comes out that suggests that patient’s program has revived potential… will the ATTC automatically reassess the case or does a physician have to re-apply the patient to n-Lorem?

Watch on YouTube:

Charissa Lipman is a part-time registered dental hygienist (RDH) and full-time caretaker/ advocate to her 7-year-old son Ryker. He received his crushing diagnosis, a variant in the CACNA1A gene, at 5mo old. Since then, life has been a whirlwind of battles, hope and relishing the “inchstones” Ryker continues to work so hard to achieve.


Hosted by: Dr. Stan Crooke.
Videographer: Jon Magnuson of Mighty One Productions.
Producers: Colin Delaney, Kira Dineen, Andrew Serrano and Amy Williford

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