ONCE UPON A GENE - EPISODE 175 Every Patient Matters - Discovering, Developing, and Providing Experimental ASO Treatments to Nano-Rare Patients for Free with n-Lorem Founder and CEO Stan Crooke Stan Crooke is the Founder and CEO of the n-Lorem Foundation. He's also the Founder and former CEO of Ionis Pharmaceuticals, t

Are you thinking about a career in genetic counseling? Maybe you are a current student or even a recent grad… Then you have to head over to our social media for a MAJOR giveaway right now! We have assembled 15 genetic counselors, including myself and some other familiar faces/voices. All 15 of us are going to be mentor

Dreams of a perfect childhood, a perfect child, and the perfect life are part of every soon to be mother’s wish list. Effie Parks did find the perfect child, but it took her down a path she never expected. Effie’s child Ford was born disabled, with an extremely a rare disease CTNB1. What followed was a path the family

ONCE UPON A GENE - EPISODE 080 Rare Disease and Grief - It’s OK That You’re Not OK with Megan Devine Megan Devine is the author of It's OK That You're Not OK, a psychotherapist, a grief advocate and communication expert dedicated to helping people face their toughest experiences. This book, her story and explanation of

Media with a Mission with Believe Limited CEO Patrick James Lynch Patrick James Lynch has to inject himself with medication every other day because his liver doesn't produce a protein that helps his blood vessels seal when they burst. He and his brother were both born with hemophilia and he's since lost his brother to

Catching and combating rare diseases, particularly those connected with genes, are huge personal battles that are truly hard to win. But how much more difficult is it for the people watching from the sidelines? Tony Martignetti sits down with Effie Parks, who shares her own struggle as a mom who fought hard for her chi