Newsletter
A Groundbreaking Gene Therapy In Record Time to Cure His Son with SPG50 Sets a New Course For Future Rare Disease Treatments with Terry Pirovalakis

Once Upon A Gene

A Groundbreaking Gene Therapy In Record Time to Cure His Son with SPG50 Sets a New Course For Future Rare Disease Treatments with Terry Pirovalakis

September 29, 2022

A Groundbreaking Gene Therapy In Record Time to Cure His Son with SPG50 Sets a New Course For Future Rare Disease Treatments with Terry Pirovalakis

ONCE UPON A GENE – EPISODE 154

A Groundbreaking Gene Therapy In Record Time to Cure His Son with SPG50 Sets a New Course For Future Rare Disease Treatments with Terry Pirovalakis

Terry Pirovolakis is a rare disease crusader and father to Michael, who has SPG50. He made a gene therapy for his son in only 18 months. The work he has done will also transform the way rare diseases are treated. He’s hosting free monthly 101, 102 and 103 gene therapy courses to help other families advance their programs. Learn more by visiting Terry’s website, cureSPG50.org or by connecting with him on social media.

EPISODE HIGHLIGHTS

Can you share a little bit about Michael’s diagnosis?

Michael was born healthy, but wasn’t hitting milestones like my other children. We learned through testing that he had a disease called Spastic Paraplegia Type 50 (SPG50). Shortly after the diagnosis, we began researching and reading articles, we flew to meet with experts in gene therapy and signed a contract a month later to start a gene therapy program which kicked off our journey.

Is there a point where it’s too late for gene therapy?

I don’t think it’s ever too late for gene therapy. My perspective is that if children can get it from 1-6 months old, gene therapy can be a cure. After that, gene therapy becomes less of a cure and more of a treatment. Unfortunately for Michael, gene therapy is a treatment and not a cure, but our goal is to cure kids by getting SPG50 on the newborn screening panel so we can cure kids- not treat them. 

What is your advice for other families who are on a journey to fund gene therapy and drug development?

Families have to understand that a lot of money has to be raised and they have to be willing to give up a lot to get the money you need. You have to have a solid family and the right team. Get your community involved, get friends and family involved and think outside the box to spread awareness about your disease. I encourage families to take my classes and reach out to me throughout the gene therapy and drug development journey.

CONNECT WITH TERRY

Website

https://www.curespg50.org/

Facebook

https://www.facebook.com/CureSPG50

Instagram

https://www.instagram.com/cure_spg50/

Twitter

https://twitter.com/CureSPG50

Email

[email protected]

TUNE INTO THE ONCE UPON A GENE PODCAST

Spotify

https://open.spotify.com/show/5Htr9lt5vXGG3ac6enxLQ7

Apple Podcasts

https://podcasts.apple.com/us/podcast/once-upon-a-gene/id1485249347

Stitcher

https://www.stitcher.com/podcast/once-upon-a-gene

Overcast

https://overcast.fm/itunes1485249347/once-upon-a-gene

CONNECT WITH EFFIE PARKS

Website

https://effieparks.com/

Twitter

https://twitter.com/OnceUponAGene

Instagram

https://www.instagram.com/onceuponagene.podcast/?hl=en

Built Ford Tough Facebook Group

https://www.facebook.com/groups/1877643259173346/

Once Upon a Gene TV

https://www.thedisordercollection.com/

You may also like

Top Health Podcasts. Delivered to Your Inbox and Eardrums.

Join Our Newsletter

Proudly supported by:

cover
Adopting Clinical Trial Technology: The Key to Success for Patients, Sites, & Researchers Adoption of new clinical trial technologies is critical to success. These innovations can lift patient burden, improve patient engagement, and increase equity and access to clinical trials.