Art’s Call to Action
Publicado el enero 11th, 20261/11/26
Nov. 26, 2025
Exciting news this week: Altay Therapeutics’ DX5057 has received Orphan Drug status from the U.S. FDA. DX5057 is currently the only oral DUX4 inhibitor in development for FSHD, which makes this an especially important milestone. Orphan Drug status gives the company meaningful financial incentives to continue advancing the program. That said, there’s still a long road ahead. DX5057 will need to move into larger clinical trials before it ever reaches the market and one of the biggest hurdles will be enrolling enough participants.
This is where our FSHD community truly matters. For those of us living with FSHD, it’s important to seriously consider participating in clinical research. If you’re in the United States, the easiest way to learn about studies near you is through
BetterLife FSHD. After completing their surveys, BetterLife will help you understand if you might be a match for relevant clinical trials in the U.S., including future DX5057 studies once they begin recruiting.
The reality is simple: the longer it takes to enroll participants, the longer it will take for this drug to reach the people who need it. DX5057 is promising. Now we just need to take the action to help move it forward.
Clearly, I’ve gotten a little too complacent. About a year ago, when I first signed into BetterLife , I was really excited. It was one platform that listed all the FSHD studies – and even highlighted the ones I might be eligible for. Unfortunately, none of them were within driving distance. I kept checking the site, at least once a month for six months, but the results were always the same – nothing close enough to participate in.
Then, just last week, I happened to learn through an FSHD chapter that someone nearby was enrolled in a study only a short drive from my house. I jumped onto BetterLife FSHD, and there it was! In fact, there were two studies nearby. I’ll admit, I was a little embarrassed – I log in regularly to complete their quick polls, so for months I was just one click away from seeing those studies.
Here’s my point: if these studies are now showing up in Iowa, they’re surely appearing in major cities across the country. We need to make a collective decision. Either we get involved to help move the studies along faster, or the science will progress much more slowly. Do we want to wait 15–20 years for meaningful relief from FSHD, or do we want to see results in five?
I’m genuinely excited about the research and the science behind it. To accelerate progress, we need people with FSHD to participate in the studies – not only in drug trials, but also in the survey-based research. All of these opportunities can be found on
BetterLifefshd.org
Right now, BetterLife FSHD has just over 600 enrolled members – a scary low number. Even with its limit of only U.S. citizens it should have much higher numbers. Clearly, many people don’t yet know about the platform. Signing up is simple: you create an account, complete a few health and symptom surveys, and the system shows you which studies you may qualify for.
As a community, we have the power to shape our own future. Let’s use it.
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If participating in the study/ research is avaliable to non american, we – 2 istaelis, a 64 years old fathet and 20 years old don both with FSHD are interested to enroll.
Contact Tzachi Heyman
Correo electrónico -ath@netvision.net.il
Phone 052-3255449.
Regards,