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UK MHRA Approves CAMP4 ASCEND + USA Today + Rare-X/Citizen Health + 6 Things #S10E219

Friday, September 11, 2026 — Week 37

CAMP4 — ASCEND EXPANDS TO THE UK

CAMP4 ANALYST DAY — SEPT. 28

USA TODAY — SYNGAP1 IN PRINT

RARE-X + CITIZEN HEALTH

SIX THINGS U.S. FAMILIES CAN DO

IEP HELP — CITIZEN HEALTH

RESEARCH — HELP WANTED

COMMUNITY QUICK HITS

UPCOMING EVENTS — COUNTDOWN

SHOOT FOR SYNGAP1 — 64 DAYS
November 14 — Hurricane, Utah
Aiming for a Cure — Shooting for Hope https://cureSYNGAP1.org/Shoot

FIGHT FOR FELIPE — 78 DAYS
November 28 — Boston, Massachusetts https://cureSYNGAP1.org/Fight

CURE SYNGAP1 CONFERENCE — 83 DAYS
December 3–4 — Denver, Colorado https://cureSYNGAP1.org/Reg26

USA 🇺🇲: Use your ICD-10, F78.A1

SOCIAL MATTERS
5,240 LinkedIn — https://www.linkedin.com/company/curesyngap1
1.62k YouTube — https://www.youtube.com/channel/UCtnPWPpqouMA_1UGOyu4W6A
43.7k Instagram — https://www.instagram.com/curesyngap1

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/

Episode 219 of #Syngap10 #SYNGAP1 #RareDisease #CureSYNGAP1 #PatientAdvocacy

Below is a transcript from the video:

[00:00:00] Hello, SYNGAP land. My name is Mike Graglia, and this is episode 219 of the CURE SYNGAP1 podcast. Today is Friday, September 11th, 2026, so we all know… where we were 25 years ago, and it’s week 37.

Big news this week: CAMP4 announced they have received MHRA, which is like the U.S. FDA, approval to have clinical trials in the United Kingdom. This is for CAMP4’s Phase 1/2 trial for CMP-002, aka the ASCEND trial, which I’m going to start calling it the ASCEND trial. So if you’re keeping score at home, Australia, they’re good to go. Argentina, they’re good to go. UK, good to go.

[00:00:40] Waiting on the EU, and we still need data on another species to apply to the FDA. Hopefully that will happen next year.

They stated in this press release, of course, link is in the show notes, that they expect to dose a human in Q4 of 2026. Check your calendars: September, October, November, December. October, November, December. Q4 is coming pretty darn quick, so buckle up for that one.

CAMP4 also announced they’re having an analyst day on September 28th. Anyone can register. Link is in the show notes. It’s from 12:30 to 1:30 Eastern Time. They’re going to talk about trial design. They’re going to talk about the unmet need of SYNGAP1. There will be some of us talking at that.

[00:01:18] And they’re going to talk about the early pipeline, which means who else might they be working on. Super exciting.

Families are like, “I want to go. I want to learn everything.” Let me tell you something. No offense to our dear friends in the finance community. An analyst webinar is painful. It’s just companies saying everything very carefully and analysts asking a lot of hard questions, questions about numbers that are way in the future. I’ve—we’ve gone to CAMP4 before and been like, “Guys, let’s just have our own webinar for families where you cover the same stuff the next day,” and they’re like, “Absolutely.” So we’re ironing out schedules. Look for that webinar soon.

[00:01:52] If you want to go to the analyst day, go to the analyst day, but don’t expect to follow much. And there’ll be a lot of talk about, like, numbers because that’s what stock analysts ask about, and who knows what the answers will be.

But honestly, I would come to our webinar the next day. We are… we are locking that in. It’s surprisingly hard to get everyone’s schedules to work, but it is going to happen.

Really excited to tell you that yesterday, USA Today took that article, which I shared with you a couple of episodes ago, and put it on the front page of USA Today. And this was about education and access to education.

[00:02:29] You remember, I’m so excited about this article because even though it was about Christina Hartman and her beautiful daughter Charlotte as the main characters, they added some context from Tony’s journey to Lake Mary. And they also… put a picture of me and Tony in there. And it was, you know, their pictures of Charlotte and their family were all professional, very good. This was, like, me and Tony taking a selfie in the back of an Uber. But, yeah, it’s just how we roll.

So check that out. If you happen to have a copy of yesterday’s USA Today, check it out in print. We did share pictures of the paper.

[00:03:00] Just doing what we can to raise awareness that our families, yours and mine, are struggling with access to fair and appropriate education for our kids. So that was a win. SYNGAP’s in the national news. Let’s keep going.

Another announcement this week was that RARE-X and Citizen Health announced that they are working together. So now this is a little inside baseball-y, but stay with me. Let’s start with PrOMMiS. PrOMMiS is our natural history study. It’s how we learn about this disease. There are clinical observations which the doctors at CHOP, Colorado, and Stanford make, and then there’s patient-reported outcomes, PROs. PROs are those surveys that you are supposed to fill in within a week of your visit.

[00:03:38] So you go to one of these sites, Penn, CHOP, Colorado, and then you… Penn, CHOP, Colorado, Stanford, and then either within a week before or a week after, you have to log on to RARE-X and fill out a bunch of surveys, and then the patient-reported data and the clinical observations go together, and we get a rock star natural history study.

Now, here’s the punchline. RARE-X used to use this other platform.

[00:04:00] To collect all the data as a software vendor, and it was just—it’s, it’s a platform. Every platform has pluses and minuses. I know the people who are on that platform. Nice people. That’s all I’m gonna say.

Citizen Health, as you know, is another platform that we use to collect retrospective natural history data. So now RARE-X has said, “Hey, given that Citizen already has all this data and already works with so many of these patients, and they have a pretty fancy interface, and they have a lot of web developers, why don’t we move RARE-X, instead of sitting on this other platform, over to sit on top of Citizen Health?” And that deal has been signed, and they are working together.

[00:04:34] And that’s super exciting for our community because now you’re going to be in a very familiar environment because you should already be in Citizen Health. You should already be in Citizen Health. It is a powerful tool to help you collect your medical records, to help… And it has Ari, which I’ve talked about and I’m going to talk about in a second.

So RARE-X and Citizen Health, congratulations to our friends over there. We’re one of the first four rare disease groups to work with Citizen Health. I have known this team for years, for years. And they are good people, and they’re working hard to help us.

[00:05:07] And I feel the same way about the folks at RARE-X. So seeing them all work together, there’s a deep bench on both sides there with parents of rare diseases in leadership. It’s going to be awesome.

Check out that press release. Sign up for Citizen. Fill out your RARE-X PROs.

Um, I’ve also talked to a number of newly diagnosed families lately, and we haven’t—they’re like, “What am I supposed to do? There’s so many things to do.” It’s not that—I really—there’s not, there are a lot, there’s a lot to do, but it’s not that complicated. But then I realized it might feel complicated, especially since we haven’t written it down yet.

[00:05:38] So we have a cool little cheat sheet, and we wrote a blog. And if you want to do something, you just go to curesyngap1.org/take-action. Slash take action. Link’s in the show notes. There’s a cool little graphic, and it lays out the six things you need to do. And really it’s more like 10 things because there’s a few sub-bullets, but it doesn’t matter. Do all those things. Work through that six-list [unclear] and take action. Help CURE SYNGAP1. Help us help your loved one have a better future. That’s the punchline.

Speaking of helping and speaking of Citizen Health, there’s a really cool webinar on October 4th, which is in about a month, a little less, about IEPs.

[00:06:15] There’s some excellent… master’s in education named Stacey Zimmerman, who’s got, like, 20 years of experience with IEPs. I’m actually going to go to this webinar. I want to hear what she’s got to say. And she’ll be talking about IEPs.

They will also, because this is being sponsored by our friends at Citizen Health, they will also talk about how to use Ari to help with your IEP. How cool is that? So you can get your IEP documents, and these documents are huge. You could upload them to Ari and say, say, “Ari, what’s changed? What’s the same? What can I get more of?” And Ari can help you with your IEP negotiation.

[00:06:44] So this webinar will be Stacey Zimmerman talking about IEPs and also Citizen talking about how to use Ari to help your loved one in the IEP. Super exciting.

Also file under super exciting: there’s a fabulous lab in Italy who was able to, through a national fundraiser, fund a two-year SYNGAP1 postdoc. I always get excited when there’s a SYNGAP1 postdoc. We fund a lot of them, but I get really excited when people find money from someone else. And this is in Rome. Who doesn’t want to work in Rome for two years? So this will be a postdoc working on iPSCs, cortical neurons, and brain organoids. Guys, we got to support this. Let’s get on it.

[00:07:22] Please, if you know any postdocs or researchers in science who wouldn’t mind living in Italy for a year, link’s in the show notes. Make sure they know about this job opportunity.

Some quick hits for the community. All right. Felipe, age three, did a Warrior Wednesday, or Wednesday Warrior one? SYNGAP Warrior? What do we call them? SYNGAP Warrior. I’m getting—guys, it’s been a long week. Anyway, Felipe’s three. Read that. It’s an awesome profile. His parents, Brian and Alana, are doing the Fight for Felipe. Good name. That fundraiser in Boston. So read that, learn about Felipe.

If you are inspired to do a fundraiser, we have a form. We got a link for pretty much everything. curesyngap1.org/fundraise. curesyngap1.org/fundraise.

[00:08:07] Go ahead, go to that link. Let us know your idea. Let us know how we can help you. We can make a lot of things happen.

Speaking of CURE SYNGAP1 helping people, Café SYNGAP Uno, which is our Spanish-language podcast, just did two episodes on parents with GRIN2A and GRIN2B. What’s cool about Café SYNGAP Uno is we not only interview SYNGAP families in the Spanish-speaking world, we’ve started to branch out to rare diseases.

[00:08:28] It’s almost like the Once Upon a Gene of LATAM at this point. It’s super cool, and I’m really proud of the team for, for the inclusion there and for reaching out to other communities and giving people voice. So if you know any Spanish-speaking rare people, check out episode 40 and 41 of Café SYNGAP Uno.

There is also, by the way, a new blog up on a recap on our Night of Impact. Ties nicely with the fundraise thing. I should have said that sooner. Guys, build a fundraiser. Helps us, helps your family, helps everyone.

Upcoming events, all in the show notes: November 14th, Shoot for SYNGAP. November 28th, Fight for Felipe. December 3rd, the CURE SYNGAP1 conference. Come to the CURE SYNGAP1 conference.

[00:09:05] Bring your family. Bring your friends. Bring your teachers. It’s going to be amazing. It was really exciting. I was in a few meetings about it today. I’m already seeing versions of the schedule, and things are changing. And it’s going to be an amazing two days. If you have a loved one with SYNGAP1 and you can be in Denver on December 3rd and 4th, trust me, there’s nowhere else you want to be.

In the meantime, make sure you’re following us on LinkedIn and YouTube. Links are in the show notes. We have a lot of followers there. Make sure you and everyone you love is there. And I said this to a family—

[00:09:35] The other day, by the way, I was like, “You know what? It’s exhausting raising a Syngapian. It’s too much damn work. And it’s even more exhausting to then update your family on everything that’s going on.”

We’re doing that work. Just make sure your family follows us on LinkedIn and YouTube and Insta and wherever they do stuff. And we’ll keep them up to date with this podcast and other relentless communication because we are relentless when we’re fighting for the future of these kids.