What’s one way to spread hope? By sharing your experiences and connecting with others who truly understand the everyday challenges nano-rare patients face. Shanna Tolbert was one of five nano-rare patient caregivers who participated in the panel, The Perilous Journey to Diagnosis and Treatment for Nano-rare Disease Patients. Listen to Shanna’s take on the importance of dialogue and relive a few patient stories as told by the parents who participated in this panel.

• 0:56 The importance of hope
• 3:38 Meeting other nano-rare caregivers and staying connected
• 6:48 The Nano-rare Patient Colloquium is intimate
• 12:41 Patients in attendance are one of the joys of the NRPC
• 16:35 Patient Journey – Connor
• 20:55 Patient Journey – Mostyn
• 25:27 Patient Journey – Lena
• 32:32 Patient Journey – Ireland
• 39:29 Comments from patient father, Luke Rosen
• 41:43 Susannah’s Story: Treated with and ASO

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.

We cannot do
this alone

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.