Q&A #1 with Patient Families

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. Question Bank: - How do you apply to be an n-Lorem patient and who do you need on your team to help? 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 - Can n-Lorem help patients who are severely advanced in their disease? 44:10 - How can treating one person can lead to breakthroughs for many more? 39:40 - What is Stan’s favorite success story from his career? 46:20