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ASCEND Trial Webinar + #SYNGAP1 Warriors, Biobank, HEOR & Industry News #S10E220

Monday, September 21, 2026 — Week 39

CAMP4 ASCEND TRIAL — WEBINAR #131

SCRAMBLE FOR SYNGAP — 12 DAYS

WARRIORS — KEEP TELLING THE STORIES

COMBINEDBrain BIOBANK — ONLY 2 LOCATIONS LEFT

HEOR — DOCUMENTING THE REAL BURDEN

HELP WANTED — PRESS

  • We have a backlog of CURE SYNGAP1 press releases.
  • Know PR / communications? We need help.
  • Volunteer skills matter just as much as fundraising.

IONIS — ANGELMAN

ULTRAGENYX — CONGRATULATIONS

UPCOMING
SHOOT FOR SYNGAP1 — Nov. 14, Hurricane, UT https://cureSYNGAP1.org/Shoot
FIGHT FOR FELIPE — Nov. 28, Boston https://cureSYNGAP1.org/Fight
CURE SYNGAP1 CONFERENCE — Dec. 3–4, Denver https://cureSYNGAP1.org/Reg26

PUBMED50 SYNGAP1 publications in 2026 — +11 vs. Week 39. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

USA: Use your ICD-10, F78.A1

SOCIAL MATTERS
5,268 LinkedIn — +28 since E219. https://www.linkedin.com/company/curesyngap1
1.62k YouTube. https://www.youtube.com/channel/UCtnPWPpqouMA_1UGOyu4W6A

Like and subscribe wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 220 of #Syngap10 #RareDisease #CureSYNGAP1 #PatientAdvocacy

Below is a transcript from the video:

[00:00:01] Hello, Syngap land. My name is Mike Graglia. This is episode 220 of the CURE SYNGAP1 podcast. Today is Monday, September 21st. We are in week 39 of the year 2026. In eight days… On Tuesday, September 29th, we will be hosting the CAMP4 ASCEND trial webinar in partnership with our friends at CAMP4. This is for families to come and learn and ask all the questions they have about the ASCEND trial, which CAMP4 is doing. This is the phase one, two trial. I told you in a previous episode that wasting your time at the… analyst call was not a good idea. This is your alternative. Come. Yuri will be there. Other leaders from CAMP4 will be there.

[00:00:39] Our leaders will be there. Learn about the ASCEND trial from CAMP4. Registration link’s in the show notes. It’s also at the top of our homepage. Please come and check it out. As usual, I want to give a massive shout-out to our dear friend, Julie Miles, and her family in Greer, South Carolina. Their fifth annual Scramble for Syngapian on October 3rd. They were on the news again. There’s a little clip in there, links in the show notes to the YouTube. Watch that, super cute. And I would say register for the event, but you can’t because it’s beyond sold out. Job, Julie. Five years of hosting your own event, building community, raising awareness for Syngap right where you live.

[00:01:16] As Miller grows older, that community is going to support him more and more. Well done. You’re an example for all of us. Hey, let’s all do that. Speaking of examples for all of us, there are three new warrior stories, and I encourage you to write a warrior story, even if you haven’t yet, even if you already have, I should say, because two of these are repeats, and they’re very good. So the first one is Weston, who is age 22. Twenty-two, can you believe it? Do you imagine not getting diagnosed until 22? That, that’s, that’s crazy to me, but it happens, and this guy was only diagnosed six months ago. Read that story. This family has clearly done a ton of work. CURESYNGAP1.org/warrior.

[00:01:56] Read about Weston. And then we also get updates on beautiful Will. Will was Warrior 21 at age nine. Will is now 16. Everyone knows that Will is Lauren’s child. Beautiful big boy. We love Will. Check him out. I see Will on work calls all the time. And then Kira’s in there. Warrior 172. Now six. She first appeared in our Warrior series at two and a half. You don’t have to know somebody to get your kid to be a warrior, guys. If you haven’t been written up, write yourself up. These stories help families connect, help people learn about Syngap. They’re really valuable. They really matter. Keep sharing them. What also matters is the biobank.

[00:02:31] CURE SYNGAP1 makes it easier for researchers to work on our disease by making are already in place in an IRB-approved, scientifically managed biobank that is administered by COMBINEDBrain, whom we are an active member of. The way we get these samples is when different members of COMBINEDBrain, and there’s about 150 these days, around the country have conferences. We’re able to go to those conferences and donate samples. There’s two meetings left that aren’t sold out. One of them is Boston 8th to 9th. That is just an opportunity for you to show up and give a sample at someone else’s conference. You don’t have to go to the conference. Just show up. Be like, I’m with CURE SYNGAP1.

[00:03:07] Of course, you should email us in advance and get all the paperwork out of the way, but that gives you a chance to donate blood from your Syngapian or their siblings. Boston 8th and 9th. The only other is, you guessed it, December 3rd and 4th in Denver, Colorado. Come to the conference. Come to the conference. I was recently told that not every single family in Colorado had come to the conference, and that makes my brain go. Guys, the conference is like a heavy dose of all the things we can do to help you. We can take samples. We can learn from each other. We can meet other families at the Denver conference. Adam’s Camp will be there, which is a Colorado thing.

[00:03:42] We have special needs dentists from Denver there. It’s going to be epic. Please, if you’re anywhere on the West Coast, you should be coming to this conference. If you’re anywhere in the country, you should be coming to this conference. But if you’re on the West Coast, this is the conference coming to you. Don’t miss this opportunity. If you’re having any issues coming to the conference, let us know. Email Lauren at CURESYNGAP1.org. I want to tell you guys, one of the reasons I wasn’t in touch last week, because the microphone’s a little close. It’s because we were working hard. We had two board meetings last week because we were approving stuff right and left and reviewing and going back and forth.

[00:04:15] There’ll be a bunch of press releases on that maybe because we’re so behind on press releases. You might not figure out what we approved last week until, I don’t know, 2028, the rate we’re going. But I’m working on that. What we did approve, though, and what you’re going to be hearing more about is the health economics work we’re doing. So we got to remember right now we’re doing the ASCEND trial with CAMP4 Four. Hopefully there’ll be other companies coming online in the year ahead. And then we’ll go phase one, two, and then we’ll go phase three, and then we’re going to have an approved drug. And then what’s going to happen is we need people to pay for that drug.

[00:04:45] So we have to start now thinking about how are we going to convince payers that this disease is burdensome enough that they should be paying big dollars for precision therapies? And that is the work we’re doing right now. We are about to launch a study of our own community. We’re going to study ourselves. We’re going to ask all of you in the U.S. to take a survey for about half an hour. This is so important to us. We’re gonna give you guys a nice gift card for doing this, but we want every family to come and tell us in plain English about the burden that this disease creates on you.

[00:05:17] This is the most important ask of the year, so I’m priming it right now. This will talk about the impact of the behaviors and the anxiety on your little one, but also on you as parents and the broader family. And there’s a couple examples I want to share because other communities have been doing this, and they’re well ahead of us. The Rett community just put something out with 323 caregivers surveyed. Links in the show notes. The Angelman community surveyed 105. These are both communities. Well, Rett has an approved drug. Angelman, not quite yet, but will soon have an approved drug. And we want to take what they’ve done and do it better. That’s how we roll. We let the big kids go first.

[00:05:55] We crib their notes and we outdo them. So check out these papers from Rett and Angelman and understand that you will have a chance to work with CURE SYNGAP1 to do the same thing in the U.S. in the months ahead. We’re going to launch this study at the conference. So hey, come to the conference. By the way, I’m making fun of myself about press releases, but I’m really not making fun of myself. We have over 20 grants that we haven’t put out a press release about. We’ve got this health economics work. Then we’ve got other things. We are not putting out press releases fast enough. And it is not because the staff is not flat out.

[00:06:25] Lauren is working like crazy on the conference. Kathryn is working like crazy on so many things. I don’t even like to look at her list. It stresses me out. Virginie’s list is equally stressful. Poor Kim is sitting there doing donations and other things and supporting everybody. And we have this ever-growing list of press releases. And it’s making me nuts because there’s so much good work I want to tell the world about, but a press release requires a little more thought, a little more attention, and a lot of project management.

[00:06:49] So if you or someone in your family has any experience with press and just likes to write and manage people and say, get this quoted, get this quoted, okay, this ready, let’s ship it. I need that person as a volunteer yesterday. It’s a short-term assignment. We have a backlog of like 20 press releases. They can crank through that in a few months and then get us on our feet. But I am desperately asking the world for help with our press releases. There’s so many good stories that we haven’t told, and I just need another warm body. Speaking of press.

[00:07:17] I spoke back in episode 217, 17, 18, 19, which was like the beginning of two weeks ago, that week that I cranked out three episodes, about the Ionis news with Alexander disease and the Ultragenyx news with Angelman disease and what was going on. Ionis since published a letter to the community about GTX-102 and about those results. It’s really worth reading that letter. Of course, I share the link in the show notes. Here’s the takeaway for you. What Ionis basically says is that their chemistry, their ASO chemistry, is really well established and has been in a lot of different trials, whereas the Ultragenyx chemistry was a little different.

[00:07:58] So what they’re basically saying is we think our drug is more potent than the Ultragenyx one, and it is a very well-established ASO, this two-prime MOE. Two-apostrophe M-O-E for those who don’t speak science. And the reason that the two-prime MOE is worth noting in the Ionis note is if you go and research the CAMP4 patent, they also use this two-prime MOE. And the way somebody very poetic said it to me is the only thing that is unique CAMP4 ASO is the business end, right?

[00:08:28] The only thing that is unique about the CAMP4 is the business end, meaning the thing that goes in and it changes the thing that regulates Syngap. Everything else is really standard. And frankly, that’s a good thing, right? We want standard, well-established, hopefully very… safe chemistry going into our kids. The only thing being different is that it’s focusing on Syngap instead of something else. So the Angelman letter from Ionis is really worth reading. And if you’re like, what the hell are you talking about? Go listen to episode 217. Also, I want to congratulate Ultragenyx, who, of course, you know, didn’t do so well on the Angelman front, but was just approved for a gene therapy for Sanfilippo syndrome type A.

[00:09:01] Sanfilippo syndrome, if you don’t know, is not good. Kids die. And this is the first-ever FDA-approved treatment for this disease. It’s a single-dose IV AAV9. We do not doing AAV9 right now. We’re doing ASO. I can talk about that later. But huge congratulations to RARE. That’s the stock ticker for Ultragenyx. They’ve been around a long time and they work so hard for the rare disease community. So I’m happy to have them see this win. Upcoming, Shoot for Syngapian, Hurricane, Utah, on November 14th. Fight for Felipe on November 28th in Boston. The conference I’ve already mentioned. Don’t miss it. PubMed is still at 50.

[00:09:36] That’s 11 more than the week, which is pretty good because we finished last year at nine more than the year. So hopefully we’ll get a few more publications out as the weeks go by, and we will have another strong year on the publication front. Thank you so much for listening. Make sure you’re following us on LinkedIn and YouTube and all the things and subscribe to this podcast. By the way, if you’re on Apple Podcasts, I would really appreciate some positive reviews.