Q&A #1 with Patient Families

April 19, 2023

Stan sits down with patient families to have a conversation and together address questions from the nano-rare community.

Amber Freed and Shanna Tolbert join n-Lorem founder and CEO, Stan Crooke, for the first ever Patient Empowerment Program Q&A episode. Amber and Shanna are two nano-rare patient mothers and rare disease advocates who have experience with the n-Lorem pathway to treatment as caregivers. Through their experiences, and by canvasing others in the nano-rare community, they’ve compiled questions aimed to help current and potential n-Lorem patients and their families become more knowledgeable about the process, receive advice, understand side effects, and more! Stan sits down with these patient families to have a conversation and together address questions from the nano-rare community.

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:

How and who do you need to help apply a patient to n-Lorem? 05:26
How does the Foundation assess cases? 05:26
Once a patient is accepted, what is the best way to track the progress of the ASO program? 12:45
Any advice for when seeking information from a research physician who isn’t communicative? 14:05
How often should a patient expect to hear communication from a research physician? 15:50
What is hydrocephalus and should patients be concerned about the side effect? 19:30
If side effects occur in treated patients, will subsequent patients be notified in real time? 30:55
What is the difference between an ASO and gene therapy? 33:55
How is n-Lorem defining an “optimal ASO”? 37:20
How can treating one person can lead to breakthroughs for many more? 39:40
Can n-Lorem help patients who are severely advanced in their disease? 44:10
What is Stan’s favorite success story from his career? 46:20

Watch on YouTube:

Amber N. Freed is fighting like a mother to cure SLC6A1. She is proud mother of adorable twins, Miss Riley James and Mr. Maxwell Norman. Maxwell was 18 months old when he received the devastating diagnosis of SLC6A1, a rare neurological disease. Ms. Freed left her career in equity research analysis and has dedicated her life to finding a treatment. If you want to learn more about SLC6A1, visit slc6a1connect.org.

Shanna Tolbert is a former teacher and co-founder of the CACNA1A Foundation. She has always wanted to be a mother and dedicates her time to caring for her two precious daughters, Ireland and Brooke, with the help of her husband, Terry. Ireland has a CACNA1A variant that causes debilitating symptoms. If you want to learn more about CACNA1A, visit www.cacna1a.org.

Credits

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

We cannot do
this alone

Together we are changing the world—
one patient at a time

We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.

We need your support

Join us on our Corps of Discovery of the mind and heart. Help us bring hope and potential help to nano-rare patients today. For free, for life.

Donate