Gene Therapy Treatment For My Disease APPROVED!!!

Added: 2026-05-11

[00:00:00]This is a very exciting video.

[00:00:04]There is a new gene replacement therapy for people ages two and older living with SMA, the disease that I have lived with my entire life. And this is a therapy that I didn't that we didn't expect for years. And it was just approved by the FDA late last year. This treatment has massive potential for so many people living with SMA, myself included.

[00:00:32]>> Yeah.

[00:00:32]>> And today we are gonna tell you all about this really exciting news.

[00:00:37]>> Yeah. So, we've talked about this treatment before. Last year, we made a video about how it could really impact our family planning journey because the treatment that Shane is on now affects his sperm. So, we were like, if this is ever approved, it's a one-time treatment and then he wouldn't be taking the medication that affects his sperm. And theoretically, we wouldn't need IVF, but like he would at least have some something to work with. So, we made a video about that.

[00:01:00]>> This was last year.

[00:01:02]>> So, essentially, there's another treatment that is kind of in the pipeline. And really, just like the timeline of these clinical trials tells us that it will be out before our child rearing days are over, most likely.

[00:01:16]>> Yeah. Although he wasn't like 100%. He was like, "I think that this will be available for you, Shane, within like, you know, the next few years." And we were just like, "Oh, that treatment does not affect or should not affect fertility."

[00:01:33]>> Yeah.

[00:01:34]>> That's like really big news.

[00:01:36]>> Yes.

[00:01:36]>> And again, like when we made that video, it felt like to me something that might come about within the next 5 to 10 years.

[00:01:44]>> Yeah. So we weren't sure if it was like going to be helpful for our family planning or you know we didn't know the timeline >> but it was not imminent.

[00:01:51]>> And this idea of gene therapy replacement already exists for SMA.

[00:01:59]There's been a treatment available for children with SMA 2 and younger and they've been receiving it and it's been showing a lot of really incredible results. kids born with my type of SMA that have received this gene therapy early on are walking as children, something that I could never even sit upright very well on my own. Um, and so it this is a a therapy that has shown a lot of really interesting and excited results in young children and now it's becoming more available for all people living with SMA. Uh, so it's exciting.

[00:02:38]>> Yeah. And I remember I think I remember the night that we found out back in November forever, but it feels her earlier in my head right now. It was similar to when I found out about the first ever treatment in that it was just a regular night and I think we were about to watch TV.

[00:03:00]>> Yeah. And then all of a sudden >> and then there was an email.

[00:03:03]>> Yeah.

[00:03:04]>> And do you remember who saw it first?

[00:03:06]Like I don't know. I think we both I feel like I I wasn't in the room and we both at the same time like saw it.

[00:03:12]>> Yeah, we did this. We like sit down before we put our show on and we both had a peruse email on social media for a minute and I think it was during that we saw it >> and I was like Hannah is this a thing that like we made a video about it but isn't due for a while.

[00:03:29]>> Yeah. All of a sudden we were like what?

[00:03:31]Second, we read the press release and learned more about it and yeah, it was supposedly going to be available within like a month or two and there was all this conversation online immediately in the SMA community.

[00:03:48]>> Everyone just being >> stunned that this happened so fast and that it was being rolled out. It was incredible. Yeah, >> I texted and called my friends and family and told them. We ran into Georgia's room and told them and it was just like >> my mom definitely was crying.

[00:04:07]>> It was I was crying. I'm like I don't get worked up very easily.

[00:04:12]>> No. And I was it was a profound >> night where I you and me were like >> how like hugging each other and it was just like wow I can't believe that this is happening.

[00:04:25]>> Yeah. So I'm going to read a quote from the press release just about like what this actually is. It says this provides a treatment with rapid onset and direct targeting of the genetic root cause of SMA. By addressing the root cause of SMA, the treatment restores SMN protein production and halts further disease progression. So you can imagine why that that's like monumental. We were elated.

[00:04:46]>> Yes. So before we tell you what happened next, first we want to taste a quick minute to thank a really special sponsor for this video. One that the SMA community will be particularly interested in. So we're going to thank them now and then we'll be back with more. Thank you to Unite for sponsoring this video.

[00:05:05]>> Yes, Unite is an amazing platform that helps patients take control of their own health journey. One of the most frustrating parts of being a patient in the healthcare system is the fact that all of your doctor's notes, medication trends, lab records, any record at all is scattered. You know, >> it's all in different places. I cannot tell you those of you that have SMA or if you're a caregiver of someone with SMA, you know, maybe if you're as bad as me, the number of times that a doctor has said to you, do you know when your last blah blood test was? And I have to give them the look that's like no and I don't even know where to find that info.

[00:05:44]>> Yeah. And not being able to find info is really really difficult. Unite is a great platform for consolidating everything into one place.

[00:05:51]>> Absolutely. And Unite has an SMA feedback program that's really cool and it's going to make meaningful changes in the way that SMA is researched and understood. And there's an opportunity right now for all of you to sign up for Unite and earn $100 simply by moving your health records there and taking a short survey. It's a great and very easy opportunity to not only take control of your health journey, but also improve SMA research and earn $100. Who doesn't want $100? I already did my survey. So, I encourage all of you. It's for people with SMA and their caregivers. So, not only is it a great platform, but a great way to improve research.

[00:06:40]>> Yes.

[00:06:41]>> To learn more and get started with the SMA feedback program, visit the link shown here or in the description below.

[00:06:47]Thank you again to Unite. And now, let's get back to our video. All right. So, how to this treatment theoretically help me in my life as well as other people with SMA. So I think the biggest thing for me is that this would be a onceanddone treatment. So I would get an intratheal injection which that means into myine >> spine which I've done before. So I'm an old pro.

[00:07:12]>> Just one more.

[00:07:13]>> But I did I did stuff it one more uh one time and then my jeans would be given.

[00:07:23]I didn't tell him I'm a scientist. I didn't tell him I'm a doctor. My genes will be given the ability to create working SMN >> protein >> protein which is the protein that I'm not currently making naturally >> which would then halt the progression of the disease where Shane's motor neurons would no longer be dying.

[00:07:46]>> Yes. And like something that's interesting is when he got the first treatment where he was getting the spinal injections, he was getting them in his neck and his arms were a lot stronger then. Um, and then now that he takes an oral medication, which is like I think diffused in the body differently. A lot of people have like less arm strength but more core strength or like more jaw strength, whatever. I'd be curious to see because this is like in your spine and it would go in that area if your arms would get stronger.

[00:08:12]like there's a chance that things that are not like your murder neurons that aren't dead yet could then be maintained and and you would be stronger in those ways. There's also a chance that like if my motor or if my SMN production were different in a way that allowed me to gain even a little bit of strength, I might be able to meaningfully exercise more in a way that would allow me to develop more muscle and increase function in ways that I can't really fathom right now. like I might be able to hold a pencil effectively again or reach my nose effectively again.

[00:08:52]>> I'm not saying that that is a guarantee or anything like that, but those are the kinds of like what ifs that a treatment like this makes me and the community has. Yeah.

[00:09:03]>> Um and so very exciting possibilities there. And then the other like main big thing for us is how it might affect our fertility journey. Do you want to talk about >> Yeah.

[00:09:16]>> all that stuff? The whole other channel learns.

[00:09:18]>> Yeah. I feel like for us that was the biggest part of this because Shane's disability is stable right now. Like his progression is stable. So it's it what came to our minds the most was like, "Oh my god, this could mean that our infertility issues are, you know, solved."

[00:09:35]>> Yeah. And it could be huge. And it's funny because it came, the announcement came around the same time that Hannah and I were separately, as you know, you saw us making videos.

[00:09:47]>> Yeah.

[00:09:48]>> Figuring out that we were ready to begin trying to have children again. Yeah.

[00:09:53]>> And we made that decision like not knowing >> that this was going to happen.

[00:09:58]>> And that this just added a really like amazing wrench into the mix.

[00:10:04]>> Yeah. like a brand new opportunity back in November that we were like, "Wo!"

[00:10:09]>> Mhm.

[00:10:10]>> Maybe this is an opportunity.

[00:10:12]>> Once that seemed like a possibility, it also made me me at least like way more >> obsessed with the idea of trying it, like of having kids, you know, because like when there were very few options, I like we could be happy either way. But like once there was like a more visible path toward or like viable path toward having kids, I was like, I will be going down that path. Like we need to head down that path right now.

[00:10:39]>> Yeah.

[00:10:39]>> Um so yeah, I think that it just speeded along or like sped along our >> our decision >> decision making about it and our excitement about it. Um once that happened, >> absolutely. It's been a weird couple of months, everyone. Uh and I think you've probably noticed that in our videos.

[00:10:54]There's been a lot of films that have been unsaid, but you can feel them in the videos. So, we're getting there.

[00:11:01]Telling you the films as we feel that we can share them with you for privacy and personal reasons and all that.

[00:11:07]>> Just the emotions around it. And you might be wondering like I'm curious if people are like the tone of this video is a little bit weird. And it is a little bit weird and it's weird making the video because this is a really really exciting thing and we want to make sure that this video is like purely about the excitement.

[00:11:22]>> Absolutely. But we're speaking in past tense and we're doing this 6 months after it was released and like that's a little odd >> and I think it's like we're going to talk about this more in another video because again we want this to just be exciting. Um but once this launched like we weren't really thinking about making content. We were thinking about like how can how can Shane get this treatment ASAP? How can we move forward with having children?

[00:11:45]>> Yeah. There's a lot going on.

[00:11:47]>> Yeah. And now you might also be wondering why we're still considering a sperm donor when it seems like that doesn't make sense with what's happening. So there's a lot more to tell. Uh but that is going to be in an upcoming video about like our experience from that point forward.

[00:12:01]>> Probably the next video, but stay tuned.

[00:12:04]Yes.

[00:12:05]>> Um we'll be sharing more very soon.

[00:12:07]>> And this is overall is a momentous breakthrough for the SMA community and we are so excited. The community feels very excited by this breakthrough and it's just a really cool thing to see all these medical advances coming to fruition. Yeah. So, yay science once again. Thank you, researchers. Yes.

[00:12:30]Still don't know how to end a video. Go science. All right, everyone. We'll be back soon with more info about how this specific treatment is playing out for us and our baby making journey. That will be very soon. Okay, bye >> bye.