FSHD Cure Initiative

03/11/2026

https://www.youtube.com/watch?v=vCzdCUdE9so

02/18/2026

02/18/2026

I’m grateful for the collaboration across this community, and for Ally’s insight and dedication in bringing this together.

01/01/2026

Happy Holidays!

A holiday greeting from kids and young adults in our FSHD community. "This disease is part of our everyday lives, and we’re grateful for all you do.Wishing...

10/29/2025

09/15/2025

We’re still looking for more subjects. If you were diagnosed with FSHD as a child and can access your pediatric records, please consider helping.

The FSHD Cure Initiative, in partnership with Solve FSHD, has launched the Pediatric FSHD Retrospective Natural History Study.
Who can join?
Anyone diagnosed with FSHD at age 18 or younger.
What’s involved?
Participants (or families) collect and share past medical records. No clinic visits or new testing are required.
Other details:
Records will be de-identified and kept private
Families may be reimbursed up to $100 for record costs
The study is observational only and has been reviewed and approved by an independent IRB
Why it matters:
This study will help researchers better understand how FSHD affects people diagnosed during childhood or their teen years. This information will help researchers design better treatments and clinical trials.
To learn more or to participate, please email [email protected]

08/27/2025

We’re excited to share a new research opportunity.

The FSHD Cure Initiative, in partnership with Solve FSHD, is launching the Pediatric FSHD Retrospective Natural History Study.

Who can join?
Anyone diagnosed with FSHD at age 18 or younger.

What’s involved?
Participants (or families) collect and share past medical records. No clinic visits or new testing are required.

Other details:
Records will be de-identified and kept private
Families may be reimbursed up to $100 for record costs
The study is observational only and has been reviewed and approved by an independent IRB

Why it matters:
This study will help researchers better understand how FSHD affects people diagnosed during childhood or their teen years. This information will help researchers design better treatments and clinical trials.

To learn more or to participate, please email [email protected]

03/28/2025

We appreciate all the interest in FSHD research, including Epicrispr's plans to begin a clinical trial. We're thankful to Amber Salzman for highlighting the severity of FSHD for those with early onset: "About 20% of cases are pediatric patients, who usually die at a very young age due to respiratory failure. Symptoms in older patients include weakness of the abdominal muscles, face, hip, and limbs as well as joint and spinal abnormalities."
This underscores the urgency for inclusion in clinical trials and treatments for all with FSHD, especially those with early onset.

The therapy, EPI-321, is intended to treat cases of facioscapulohumeral muscular dystrophy, a genetic neuromuscular disorder.