Cure SPG50

02/04/2026

What amazing and exciting news for our SPG50 community!

New SPG50 Gene Therapy Trial
Interventional / Gene Therapy Study

This clinical trial is enrolling participants in an interventional (gene therapy) study for individuals with SPG50. Enrollment into the treated arm is limited, protocol-defined, and site-specific.

How it works
📋 1 gene therapy treatment delivered by a single spinal procedure
📋 Follow-up visits to monitor health and development over time

Important information
🔹 Treated-arm enrollment is limited
🔹 Eligibility is determined by the clinical trial protocol
🔹 Additional study sites in Italy and Spain are enrolling soon

📩 Email Please contact [email protected] for questions about trial status and eligibility. More details can be found here https://lnkd.in/gS9g2ssQ

🔗 Register with Citizen Health to receive updates about future studies and https://lnkd.in/gyVaGCQq

07/30/2025

Dear AP4 community,

CureSPG50 has partnered with Citizen Health!!

We're calling on all the AP4s community to sign up to Citizen Health today!

https://www.citizen.health/partners/curespg50

Citizen Health is an incredible (and FREE!) platform that helps you keep all your child’s health records in one place and track your child’s data secure, organized, and always accessible. This includes appointments, therapies, or studies your child has been part of, Citizen makes it easier to manage everything in one spot and you can access the data free of charge.

Why is this important? Because it helps us:
Know who you are…
Keep you in the loop when new studies are launched and potentially include you in our upcoming trials..

This is your chance to contribute directly to research while also benefiting from tools that support your own health journey.

Spread the word… the more AP4s who sign up, the stronger our data and the better our studies!

CureSPG50 has partnered with Citizen Health to help patients and families use their health data to improve care and drive groundbreaking treatments.

09/17/2024

Over the last six years, there have been some incredible people working to save children like my son and others facing devastating diseases. These are the people I consider heroes, and today I was fortunate enough to see three of them.

Dr. Juan Bonifacino: He and his team were among the first to research SPG50 and the AP4 protein complex.

Dr. Raffaella De Pace: She played a key role in testing the therapy we developed, even going in during the height of COVID to keep things moving forward.

Dr. Steven Gray: He created the therapy for Michael and others, and works tirelessly to bring hope to families like ours.

Thank you for being the heroes we needed.

Steven Gray, De Pace, Bonifacino

08/30/2024

An amazing article by Fox News on helping save another children affected by SPG50 like Michael.

Unfortunately I cannot share this news article but if you type in "Foxnews SPG50" it will come up.

If you can please donate to their GoFundMe, Our Charity or our GoFundMe, these kiddo's deserve to live better lives!

Their GoFundMe: https://www.gofundme.com/f/37g0ef11i0?qid=cc876570cdc5b3be8cfed3abea40279d

Our Charity: http://CureSPG50.com

Our GoFundMe: https://www.gofundme.com/f/cure-michael-curespg50-spg50?qid=cc876570cdc5b3be8cfed3abea40279d

We're the Lockard family, and we are fundraising to help cure both of our children, and al… Rebekah Lockard needs your support for Naomi and Jack Battle SPG50

08/28/2024

Many of you know about my wild quest to create a therapy for Michael, and it all kicked off with a bootcamp at Ultragenyx. If you're a parent of a child with a rare disease and are determined to develop a therapy, I can't recommend anything more highly than the Bootcamp at Ultragenyx, happening November 12-14.

Space is very very limited to sign up quickly.

https://app.smartsheet.com/b/form/ba38b887c7b1402e87deca3a8056c6a4

08/05/2024

Thank you Kevin Jiang at The Toronto Star for sharing our story. We have an amazing community, friends and family that helped give Michael a better life!

Now we need to save all the rest of the kids and eradicate this disease!

Two-year-old Michael Pirovolakis was diagnosed with a devastating illness. Four years and $4.5 million later, researchers made a breakthrough.

06/28/2024

I want to thank The Hospital for Sick Children for saving my son and truly going above and beyond. We moved mountains together, and hopefully, the treatment we created for Michael will turn out to be a legacy for others with rare diseases, proving that nothing is impossible!

The findings from SickKids’ first single-patient gene therapy trial show promise in halting the progression of SPG50.

06/05/2024

Today was an incredible day—I had the honor of speaking at the Veeva conference in Madrid, addressing an audience of over 1000 leaders in the biotech field from around the world. It was a humbling experience to share my story and connect with such an amazing group of attendees. I truly believe that each and every one of them plays a crucial role in developing treatments that impact us all.

A huge thank you to Chris Moore, Rik Van Mol, Laurie Wood and everyone involved in making me feel welcome at the conference.

Together we can and will give children living with rare disease a better life!