Feb 01 2024 40 mins
In our last episode, Nate Plasman shared his story about his little boy’s diagnosis with a rare disease and the battle to get him into a clinical trial. Today, we’re looking at what can happen once that drug is approved by the FDA and doctors can begin to prescribe it.
I’m talking to 2 families who live on opposite sides of the country, but were brought together in the fight of their lives. For so many of us facing the unimaginable – a loved one battling an illness or disease for which there is no cure – it seems that an approved treatment could be the answer. But oftentimes, that approval is the beginning of another heart-wrenching fight. The fight for access to that treatment.
Our story today focuses on the June 2023 FDA approval of Elevidys – the first approved gene therapy for 4 and 5 year olds with Duchenne muscular dystrophy. Heather Hay, and Phil and Jena Huber share their families’ experiences, their deep commitment to helping other children get access to a treatment, and their fierce belief that a community can drive change.
This is the individual and unique experience of these 2 families and our focus is not so much on the treatment itself as it is on what families go through and how love can move mountains.
I’m talking to 2 families who live on opposite sides of the country, but were brought together in the fight of their lives. For so many of us facing the unimaginable – a loved one battling an illness or disease for which there is no cure – it seems that an approved treatment could be the answer. But oftentimes, that approval is the beginning of another heart-wrenching fight. The fight for access to that treatment.
Our story today focuses on the June 2023 FDA approval of Elevidys – the first approved gene therapy for 4 and 5 year olds with Duchenne muscular dystrophy. Heather Hay, and Phil and Jena Huber share their families’ experiences, their deep commitment to helping other children get access to a treatment, and their fierce belief that a community can drive change.
This is the individual and unique experience of these 2 families and our focus is not so much on the treatment itself as it is on what families go through and how love can move mountains.