Saturday, December 20, 2025 – Five days till Christmas, 11 days left to raise funds to CURE SYNGAP1
AES was exceptional in many ways, here are a few:
Rare & SYNGAP1 were both very visible, posters with our Logo and names of staff were seen! Posters: https://www.linkedin.com/posts/graglia_syngap1-curesyngap1-activity-7408291479187755008-rMru
Our conference was standing room only and had investors! Even got a mention in their research report! https://www.investing.com/news/analyst-ratings/cantor-fitzgerald-reiterates-overweight-rating-on-camp4-therapeutics-stock-93CH-4403281
ProMMiS Launch was a massive win for patients. Collaboration.
Praxis and Lundbeck recruited for exciting drugs and CAMP4 talked about their ASO and recruiting next year.
Our community’s presence was felt well into AES.
Aaron’s post on growth! https://www.facebook.com/aaron.j.harding.5/posts/pfbid0231DtMVUtkZa4eXLv8C8qbf4xEN95aRP1xJ8sGNNvun7aDuUyZVatMWUjjigdXfg1l
Pre-register now for Denver: cureSYNGAP1.org/Pre26
Fundraising. We are YTD $1.68M which is below $1.86M in ’23 and $1.97M in ’24. We need to really double down on fundraising for the next two weeks and into next year. Support our campaign at cureSYNGAP1.org/Unlock
ACTION ALERT 🚨Call Bernie and urge him to help children at NO COST to the USG by supporting the PRV. (202) 224-5141
https://www.linkedin.com/posts/christine-waggoner-71b1555_mibagents-makeitbetter-osteosarcoma-activity-7408186315592085504-A6AY
PUBMED is at 58 for the year, that is +4 over our best year, last year. 187 since 2022, more than half of our SYNGAP1 Knowledge (365) has been created in the past 4 years!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.1998-2026&timeline=expanded&sort=date
SOCIAL MATTERS
4,519 LinkedIn. https://www.linkedin.com/company/cureSYNGAP1/
1,490 YouTube. https://www.youtube.com/@cureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/cureSYNGAP1/
$CAMP stock is at $6.38 on 19 Dec. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 192 of #Syngap10 #CureSYNGAP1 #Podcast
Below is a transcript from the video:
Mike Graglia
Hello, SynGAP land. My name is Mike Graglia. Today is Saturday, December 20th, year 2025. This is episode 192 of the SynGAP, CURE SYNGAP1 podcast. It’s been about a month since I’ve done a podcast because the last one I did was right before we went off to AES. AES, the American Epilepsy Society. It’s a huge annual meeting for neurologists and SRF. I mean, CURE SYNGAP1 very strategically has our annual conference right before AES so it’s like Thursday, Science Day where the clinicians and the scientists can come. And then Friday, they can jump right into AES. When they’re jumping into AES, we have family day on Friday. So it feels like, hey, 2-day conference, Mike, why are you so tired? Well, let me tell you. We flew out on Tuesday. Dinner on Tuesday night at Suzanne’s house. All-day board meeting on Wednesday. A lot of strategy and planning for next year. Wednesday night. Reception with the speaker for Thursday. Thursday, huge science conference, wall to wall. Incredible meeting. Not only did we have what we’ve had for the past four years, which was companies and families and researchers in the room as well as clinicians. But this year we also had investors joining us. These investors were covering CAMP4 because CAMP4 has committed publicly to bringing a candidate into humans in the second half of 2026. So investors are paying close attention and they’re like, ‘Hey, let’s go check out this conference.’ And I got to tell you, they gave us some really, really good reviews. They gave us some really, really good reviews. And they wrote an investment report on CAMP4 that actually profiled the SYNGAP1 meeting. I don’t know if this is the first patient advocacy meeting that has had its own equity research report dedicated to it or not. But if it isn’t the first, it’s one of the first. And it’s a really exciting report. I wish I could read it to you, but it’s long and I’m not supposed to share it. But there is an article about it in investing.com and I share that link in my notes. Check that link out, guys. It’s pretty exciting that these people are talking about it. But anyway, back to my thing. Tuesday, all-day board meeting. Tuesday, fly in. Wednesday, all-day board meeting. Thursday, science day. Friday. Family Day. And by the way, Thursday night for all the families coming into town, we have a big rare reception. Which was us, SLC6A1, GABA-A. MED-13L, Citizen Health, CombinedBrain, other companies. Co-sponsoring a big reception. Which is just a lot of fun. And of course, our family is very convenient for our families. They get to go Friday family day. And then family dinner, which is exciting. And while that was happening, for the first time ever this year, we had our promise meeting. So we, every quarter, get together with the clinicians from my natural history sites, currently CHOP, Colorado and Stanford. Make sure you’re taking part in the Promise Natural History Study going to one of those sites. As well as companies who want to know what’s going on. So they sponsor some of those sites so they can learn about what we’re learning in the natural history study. Why do companies care about natural history study? Because the natural history study dictates what we should be measuring in a clinical trial. That is huge, and I got to tell you, I was already tired and exhausted at the end of Thursday but when I got up on Friday morning, while you guys are having family day, and I kicked off that ProMMiS meeting. I looked up and I saw clinicians from CHOP, Stanford, Colorado Children’s. And I saw these companies and I saw our leadership, Marta, Virginie, et cetera, were in the room. And I was like, holy smokes, this is happening. This is real. These are people, I mean, anyway, I don’t want to belabor the point, but it was huge. It felt like a silent milestone, right? Like a really big step forward for SynGAP and some of you didn’t even know it was happening, but it happened and it was great. The other thing about AES and our meeting that I wanted to talk about was Rare was very visible. I think a couple of years ago, you go to AES and AES is like all epilepsies, right? As recent as a couple of years ago, people were just talking about general epilepsy and big epilepsy meds and whatever. And rare diseases were like, what about us? What about us? Genetic cause of epilepsy. Increasingly over the past few years, you’ve seen us get, not dominate the conversation, but get a lot more airtime. And I would say rare disease generally, and SynGAP specifically, certainly was covered at the meeting, just by posters. I mean, SynGAP was mentioned in 17 posters this year. So there’s presentations and big conference-y stuff, but also there’s huge poster sessions where people share their emerging research. And 17 posters on SynGAP is not a small number. The other thing that was cool is we give these beautiful lanyards for our meeting, right? So you get your name tag on one of these lanyards for our meeting. And then you go to AES and they give you these boring lanyards that just color code you into different sessions. There were a lot of people, myself included, of course, wearing these lanyards around AES, kind of showing their SynGAP pride, showing their colleagues, yeah, I went to this amazing meeting. Let me tell you what happened. That is exactly, guys, why we do the Science Day right before AES. It’s not because the first Thursday in December is a convenient time for a meeting. Not at all. But, for us at least. But for the clinicians who are going to AES anyway, it’s a really convenient time for a meeting. And we really make the most of it because what we need to get better care for our kids is clinicians who understand and are talking about SYNGAP1. So anyway. We’re very visible. Check out the posters, links in the show notes. Our conference was standing room only. We got covered in equity research. Very exciting. The promise meeting launched. We had people recruiting for our kids at the meeting. So hopefully at our meeting next year, you’ll have CAMP4 recruiting for clinical trials. But this year, we had Praxis and Lundbeck recruiting for small molecule. So remember, CAMP4 is working on RNA, which is, I mean, ASOs, small piece of RNA, specifically targeted at SynGAP. Whereas Lundbeck and Praxis this year were coming at us with small molecules for epilepsies, but the inclusion criteria, the way you get into the trials, is you have a DEE. In the old days, it was like you have Dravet or you have LGS. Dravet or LGS, and everyone else can wait. Finally, people have realized, wow, there’s a lot of people who don’t have Dravet and LGS who still are suffering tremendously. And now… Companies like Lundbeck. formerly known as Longboard or Lumbeck Longboard and Praxis are now saying, hey, we have this molecule. We think we can help all your kids. Please take part in that trial. So. When the videos come out, watch those videos of the great talks from Lundbeck and Praxis. But also on our website, if you go to clinical trial, just go to search type clinical trials. Both those trials are there. If your child has countable motor seizures, countable motor seizures. So not the classic SynGAP, eye roller, eye flutter, absence, but you can see them seizing. Please consider taking part in those trials. Very, very interesting work. And of course, we also had CAMP4 present. And next year, hopefully, they will recruit. So our presence was felt, guys. It was a great success. And another way to think about the growth is Aaron Harding, one of the CURE SYNGAP1 OGs, did a post showing our first conference and our most recent conference. And the change is obvious. Net-net, if you weren’t there or if you were there, either way, time is now. Pre-register for Denver. Put it in your calendars. Plan on coming to Denver. First Thursday, Friday of December next year. It’s going to be amazing. It’s going to be amazing. If this year, we’re hockey sticking, guys. I mean, it’s… I’m almost nervous about how much is going to happen at this time next year. So that’s Denver. Fundraising. It’s all very exciting. Nothing is free. We have to raise money to get this work done. Year to date, we have raised $1. 68 million. That sounds like a big number. We ended 23 with $1. 86 million. We ended 24 of $1. 97 million. We need to keep going up. Going down is not an option. Our kids deserve more, not less. So we are at $1. 68 million. We have 11 days left in 2025. Please make sure you’ve given your donation to CURE SYNGAP1 this year, go to our website: curesyngap1.org/donate. Take part in our year-end campaign: curesyngap1.org/unlock. For Unlock their tomorrow. And tell your friends and your family, hey, this organization is incredible. There are three paid staff who are underpaid. There’s this one crazy guy, Mike, who leads it. He doesn’t even take a salary. And all we’re trying to do is get better medicines for our kids to improve our futures. Please support CURE SYNGAP1. That’s your pitch, guys. Give the pitch, give the pitch. We need it. The other thing we need is priority review vouchers. Um, if you if you don’t know what a priority review voucher is, basically, to give companies working on like tropical diseases or rare diseases like ours an incentive. The U.S. Government used to give drug companies who got a drug for a rare disease a priority review voucher. And this would make your FDA time go from… 10 months to 6 months or something. For a small rare disease company, that kind of matters. For a very large company, that can mean big dollars. So what happened is: big companies would buy the priority review voucher, the PRV, from little companies. And they would get through the FDA faster. Here’s what you need to know. This cost the government nothing. This helped companies invest in rare diseases. The fact that this is currently stopped, there is literally an act called the Save, Give Kids. Chance act give kids a chance. Act and one senator. Guys, one senator stopped this from passing. Bernie Sanders, who I’m generally pretty aligned with, but I think we need to all call Bernie and be like, ‘Bernie, this is is not okay.’ Approve the PRV, the Give Kids a Chance Act. Let us have a PRV. Please, Bernie, my child has a rare disease. We need the PRV so companies will continue to invest in rare disease. Bernie’s number is the capital switchboard, 202-224-5141. Call them. Right now, just while you’re listening, hit pause, dial 202-224-5141. Say, ‘I want to talk to the office of Senator Sanders and leave a message that the priority of your voucher is important.’ Your child is suffering and they deserve better. I want to finish on a high note and I got to move fast. PubMed, guys, is at 58 publications for the year. You’ve heard me talking about PubMed. That is the most publications on Syngap in any year ever. We’re four over last year. That’s 187 publications since 2022. Why does that matter? It means that in the past four years, in the past four years, we’ve seen more. We’ve seen half of the publications on SynGap happen. So the knowledge around SYNGAP1, half of it has been created in the past four years. That is incredibly exciting. Incredibly. Exciting. We are making progress. Continue to support CURE SYNGAP1. Continue to stay tuned. See you at the conference next year. Thank you so much for listening. Like and subscribe to podcasts wherever you follow.