Anyway, any progress in treating similar conditions is great news.
"These kind of genetic therapies seem to reinforce this idea of deafness being a problem in need of eradication, and that the only solution for disabled people to fully assimilate into society is through a medical intervention," says Jaipreet Virdi
- Loss of hearing
- Identity built around loss of hearing
I'd also point out that creating an identity around a feature of one's body is a poor man's substitute for loving yourself. No wonder that people who do that get so defensive. Everything becomes a personal attack to them. While it's understandable, it doesn't make it any smarter, wiser, or functional.
I’m a competent, highly functional person. I also have idiopathic hypersomnia and IBS-D. I’d love a fix for either; I want to live the best life possible.
The whole deaf community opposition to treatment reads as just a defensive mechanism. Being deaf means that one of your limited amount of senses doesn’t work. By definition, they’re disabled. That’d be like people whole are really near or farsighted not using glasses because they’ve decided not being able to see is their culture or personality. It’s ridiculous, and that viewpoint should be more than ridiculed when deaf parents don’t pursue treatment for their children.
I'm not deaf, but I'm legally blind and autistic. Interestingly, I've never once heard of someone take this position with regards to visual impairment. Why is that seemingly so universally agreed upon to be a "real" disability, and things like autism and deafness aren't?
1. You can only be whole ifwe heal you 2. I am fine as long as the world doesn't make me suffer
I can see both sides, but I also see that 1 is incredibly condescending.
(blind user)
would that ACTUALLY happen, though? I challenge that assumption.
Take this hypothetical scenario: magic... magically all deafness is gone, suddenly and instantly. Would this destroy friendships? Would this erode personal relationships? Would this destroy (the very useful invention of) sign language? Would this destroy books or media? Would this devastate financially members of this community? would this kill anyone?
Well, besides the secondary effects of suddenly hearing, potentially leading to accidents. Do you actually think any of the above would happen?
I don't actually see anything like that happening. This is conservatism dressed up wearing a minority's hat. This is staunch resistance to change because of fear of lacking the familiar experience using a gross comparison to prevent reasonable analysis.
But I also believe in personal choice. Mandating conversion is not a power I want to give the government in any capacity. I just do not see the 'genocide' argument.
This is an example (like Christianity) about how horrible ideas attach themselves to identity to prevent their excision from their host. If you don't think Christianity is a good idea: suddenly it's a personal affront to them. If you don't think being deaf is an advantage or neutral: suddenly it's a personal affront to them. Be wary of anything attaching itself like this to your identity: you usually get infected when you are too young to have defenses.
^: https://www.npr.org/2026/04/23/nx-s1-5795526/deafness-gene-t...
My wife and I carry mutations to the gene so we’ve done preimplantation genetic testing to select the embryos that haven’t been affected and our daughter can hear just fine! We have enough unaffected embryos that we can have another child but if we can have a third we’d probably want a boy[0] and both of our male embryos are coincidentally affected. If somehow we’ve managed to delay long enough for the corresponding Decibel TX AAV.103 gene therapy to come to market, then this will be an incredible triumph of modern science and technology over nature.
Here’s hoping!
If you’re curious about this process, I’ve written about it here: https://wiki.roshangeorge.dev/w/IVF
And here’s the treatment pipeline image I nicked off decibel TX’s website before they were acquired a year and a half ago https://wiki.roshangeorge.dev/w/File:Screenshot_Decibel_Tx_P...
I’m super thrilled everything has gone through so fast.
0: it would just be nice to have children of both genders; a weak preference - if I have 3 daughters I would be thrilled anyway
Unfortunately, I have seen treatments arrive for almost every type of deafness except the one affecting me. I contracted the mumps at a very young age, approximately 5 months old, and I now have nerve deafness in one ear making me completely deaf in that ear. The ear drum still operates, as I feel pain in that ear when around extremely loud noises. I absolutely do not want a cochlear implant, so it seems I may be stuck like this for the rest of my life. :-(
Otoferlin [1] uses calcium as a cofactor. These mutations happen for a reason. The enzyme is not only located in the ear, but also in the brain and bone marrow [2].
Will there be repercussions if the virus leaves the local area when the therapy is injected?
These OTOF mutation have their highest expression in the Turkish population. Many people with other variations of this gene only experience deafness when they have a fever[3]. So in my opinion, I would like to see ten year outcomes before celebrating.
[1] https://www.uniprot.org/uniprotkb/Q9HC10/entry
[2] https://www.proteinatlas.org/ENSG00000115155-OTOF/tissue
[3] https://www.frontiersin.org/journals/cell-and-developmental-...
https://youtube.com/shorts/O8HZw4AbttQ?si=QSCjyDGEIr3Ll-5h
My short interpretation is that you basically inject a virus to mutate cells permanently and it regrows missing hair that takes sound waves and turns them into electrical impulses in the brain.
Absolutely amazing!
Often times these are rare disease, even though there are some like type 1 diabete, and so they have very little research on them but the gene responsible for the disease are known.
Thank you.
> The biotech company will also offer the first hearing-loss gene therapy for free to eligible U.S. patients following regulatory approval of the product earlier Thursday.
https://www.cnbc.com/2026/04/23/regeneron-inks-drug-pricing-...
Does gene therapy treatment correct the issue just in the individual, or does it also correct the genes inherited by the individual's children?
That might also suffice for sperm, which are constantly being created anew, but I'm not sure that would work for eggs, which are largely formed in gestation. But there might be an egg-specific variant treatment.
I thought public policy was avoiding this specific thing for this specific reason, but I’ve been admittedly out of the loop
Certain kinds of things like trisomy-21 (Down's Syndrome) are actually routinely screened for via things like the Maternal Serum Alpha-FoetoProtein test (primarily used for neural tube defects) or nuchal translucency (thickening could mean trisomy-21, trisomy-18, or other things), so you will know if your embryo likely has these things.
Things like this are key to countries like Denmark having a very low rate of people with Down's Syndrome since nearly all pregnancies that test positive for these are terminated.
So because many of these are routine, they're effectively free at the point of service for many pregnancies (covered by insurance) but the more advanced things require money. I have the costs laid out on the page but the test was roughly $2500/embryo for the test once the IVF lab did their thing. The IVF service was full service and $25k but we went with a middlingly expensive service.
But, ignoring this, you wrote:
> If somehow we’ve managed to delay long enough for the corresponding Regeneron AAV.103 gene therapy to come to market, then this will be an incredible triumph of modern science and technology over nature.
How does this relate to offspring exactly? The "delay long enough" part makes no real sense to me. Also, no treatment is usually always 100% effective, so I don't understand the "delay long enough" part either. Plus, there is no "nature" anymore than there is a divine being. What this here is simply the difference between having technology; and not having it. "Nature" is not part of any equation here other than regular genetic information and how it is changed, "naturally".
That’s about when I found out about the Decibel Tx therapy ongoing. If I have any underlying motivation it’s that maybe we could get earlier access to the therapy. Sadly, scientific studies don’t admit fan clubs preferentially.
The delay long enough is that we have all of our embryos in cold storage and we’ve been able to select the ones that are unaffected. My daughter is a bit over a year old and we’re planning a second implantation in the next few months where we will obviously select an unaffected embryo. But a third child, if my wife wishes, will be another 3 years from now. If PGT allowed us to go from a 2023 plan to have kids to a 2029 son born from a male embryo we were able to keep in cold storage till a therapy was available to give him hearing at birth, I think that qualifies as being able to delay long enough.
And about the Nature comment, imagine it a form of metonymy. I’m just referring to random chance and genetic mutation and so on. I’d call my glasses a triumph of man over nature too :)
the pedantry of some HNers never ceases to amaze me. all of these words to not actually say anything.
https://investor.regeneron.com/news-releases/news-release-de...
That of course doesn't rule out problems, but the treatment doesn't naively turn on the protein production either.
In terms of 'leaving the local area', there was a recent treatment intended to be done on one eye first, just in case it did not go as planned. It spread to the other eye:
https://www.cam.ac.uk/research/news/gene-therapy-injection-i...
Viral vector DNA was detected in the anterior segment, retina and optic nerve of the untreated eye. The unexpected visual improvement observed in the untreated eyes could therefore reflect the interocular diffusion of rAAV2/2-ND4. Further investigations are needed to confirm these findings and whether other mechanisms are contributing to this bilateral improvement.
Seeing as the eye was directly injected, it's unclear how it spread. Blood, likely.
With regard to your point about shedding or systemic exposure. If non functional copies of OTOF in the other tissues expressing the gene were to be replaced by functional copies, what is the concern? How would that negatively impact patients? Doesn’t seem like this would/should be a concern.
Also, mutations don’t have to be teleologically beneficial to occur and persist. They can persist because they are not fatal nor do they impair reproductive competence.
Personally I think I would want to hear once in my life, even if it meant a potentially shorter life.
I'm glad the Trump admin prioritized this.
Primarily the focus has shifted to faster approvals with evidence for new methods and drugs coming down to one high quality trial, and removed stipulations for randomized control trials for ultra rare diseases.
Could you share how RFK's policies helped bring this to market faster? (Not challenging you, by the way. Just need help connecting the dots.)
Nuance is fine. There's not much nuance in the parent post.
> Today’s approval was issued 61 days after BLA filing, marking the sixth approval under the Commissioner's National Priority Voucher (CNPV) pilot program and the first gene therapy product approved under the program
That appears to be a late 2025 program based on the dates on the FDA page which would make it a Trump administration policy change. I don’t have any real knowledge here though. I’m just a guy clicking links. But if this is a policy change that’s exciting for approval of the other gene therapies for related conditions once the technology is solved.
0: https://www.fda.gov/industry/commissioners-national-priority...
Latest in this area: https://www.fda.gov/news-events/press-announcements/fda-laun...
“President Trump promised to accelerate cures for American families — and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,” said Health and Human Services Secretary Robert F. Kennedy, Jr. “We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most.”
“This guidance is a critical step the FDA is taking to tailor our regulatory approach to patients with ultra-rare conditions,” said FDA Commissioner Marty Makary, MD, MPH. “It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases.”
Now, you can be caught in the fun partisan civil war on emotions or simply look at outcomes. Good work being done at the FDA, just fact.
And, let's not forget that RFK said: “every Black kid is now, just as a standard, put on adderall, [selective serotonin reuptake inhibitors], benzos, which are known to induce violence. And those kids are going to have a chance to go somewhere and get re-parented, to live in a community where there’ll be no cell phones, no screens, you’ll actually have to talk to people."
So yeah, according to RFK, every black kid in America deserves "re-parenting". He should resign today.
AFAIK critics pointed this to be a form of regulatory capture as well.
At the risk of turning this positive thread negative, there exist deaf people who would deny their children the opportunity to hear, and very little makes me more furious than society's willingness to tolerate this form of child abuse.
There's also an issue that, assuming they work similar in this regard to cochlear implants, the treatment has to be performed at a very young age before its possible for someone to consent or choose whether they want to be part of the deaf community.
Here's my test: If my child was deaf and asked me when they were old enough to know that I declined to have them treated based on these arguments, I cannot even imagine them being okay with that.
I suspect so.
https://en.wikipedia.org/wiki/Endangered_language
I'll also provide my favorite "it won't happen to us!" example. https://en.wikipedia.org/wiki/Association_of_German_National...
I however do not wish children to be subjected to the will of the parents when that will dictates that the child's variety of sensory experience is intentionally limited by withholding medical intervention. That is cruel.
There will never not be deaf people, and we should build society so hearing isn't a requirement.
Good faith, bad faith ... does not matter. Decisions and changes are public record. Read for yourself.
A good outcome doesn't mean there was good work being done. You are conflating the two. You can make bad decisions and still have a good outcome.
I'm happy that this looks like a good outcome, but the current FDA is doing terrible work.
RFKs deleterious impact on scientific research and its funding is well documented in the context of the NIH. 2025's Bethesda Declaration ably details the culture of 'fear and suppression' present under RFK, and the $9.5bn in grants and $2.5bn in contracts he had cut, impacting over 2,000 projects. It concludes with a chilling warning regarding plans to cut up to 40% of NIH's $48 billion budget in the future.
https://en.wikipedia.org/wiki/Bethesda_Declaration
At an FDA level, the same strategy was clearly evident last August when Trump fired CDC Director Monarez after clashing heads with RFK over vaccine policies barely a month into her role. Kennedy had demanded she fire career agency officials and commit to backing his own advisers. Four high-ranking officials resigned in support with Monarez.
In a similar vein, RFK then performed a clean sweep of the legacy 17 person vaccine panel in favour of his handpicked eight person vaccine panel – half of whom share ideologue Kennedy’s famous distrust of vaccines. Democrats on the Senate Health Committee summarised it blunty in an open-letter to RFK: “By removing all 17 of ACIP’s members and replacing them with eight individuals handpicked to advance your anti-vaccine agenda, you have put decades of non-partisan, science-backed work – and, as a result, Americans’ lives – at risk."
Moderna CEO Stephane Bancel subsequently said the company would not invest in new phase 3 infectious disease vaccine trials due to growing opposition from U.S. officials to immunizations.
This was then further compounded when the FDA RTF'd Moderna's new Flu Vaccine on spurious grounds in February, with the Alliance for mRNA Medicines calling the decision “unprecedented,” claiming the FDA was in “disarray,” and warned of a “threat to public health.”
Even last month a federal judge concluded RFKs actions re: the panel were not lawful, and that earlier votes by the panel to downgrade recommendations for hepatitis B vaccines for newborns and COVID-19 shots were invalid, blocking the Trump administration’s much publicised overhaul of the childhood vaccine schedule.
The only partisan stance at the moment would be not acknowledging the systemic dismantling of these scientific safeguards and institutions to the detriment of the American population as a whole.
How about https://www.fda.gov/news-events/press-announcements/fda-elim... ? Game changer for RWE. Not Science?
Or this one: https://www.fda.gov/news-events/press-announcements/fda-acce... ? A call for more research on certain compounds. Not Science?
I know the MSNOW daily hate machine is fun, so engaging and gives life meaning. But laying off the emo crack for a while would open your mind beyond partisan viewpoints.
Sign language is exactly as rich a linguistic and cultural tradition as all vocal languages combined, it is an equal branch of human expression & life. It's not the hearing or deafness exactly, it's the experience of being one of the participants in and caretakers of this tiny but vibrant and important domain of humanity.
More crucially: Cultures do not have a right to exist. If my culture thriving is straightforwardly at odds with society's physical wellbeing, I need to change and adapt, not society.
- Sign language is not rich in importance and tradition
- Sign language is not an equal branch of expression and life
- Sign language, and body language, are not important and have no profound implications
> makes it impossible to dismiss as merely failing to love themselves. (??)
I would encourage you to practice reading with comprehension. I said that building identities around features of one's body is a poor man's substitute of self love. If you don't understand what that means and how it differs from "dismissing deaf people's language as their failure to love themselves", let me know, I will try to explain.
> It's not the hearing or deafness exactly, it's the experience of being one of the participants in and caretakers of this tiny but vibrant and important domain of humanity.
Great, at what point did I suggest that any of that is unimportant, prohibited, unworthy of continuing etc. etc.?
I called this statement stupid:
These kind of genetic therapies seem to reinforce this idea of deafness being a problem in need of eradication
and that the only solution for disabled people to fully assimilate into society is through a medical intervention
There, that's what I said and meant.
As an analogy, how would you feel about a new mandate that all babies learn English as a first language?
I think it would be wonderful in its effects (I am not a native English speaker), but I don't like the "mandate" part.
As for the other point you are making - the language and culture were developed to work around physical issue of not hearing. Those who have learned the language can continue to use it after regaining hearing. I don't see why those who can hear couldn't learn it if they wanted to (e.g. to communicate with someone who decides to not pursue treatment for whatever reason). I also don't see why preserving something, that solves a problem that now has a better solution, is so important.
When it comes to children, then, the question is not just "do I want my child to hear better than I can", but also "do I want my child to speak the same language and belong to the same culture that I do" - something most parents want very much.
That's simply: 'what is best for my child' vs 'what is best for my relationship with my child'. Only one of those actually has the best interests of the child at heart. Only one of those opinions is respectable. Growing up with the latter leads to resentment towards the parent generally.
Since you are very eager to police what I can or cannot do, let me return the favor: you can stop projecting beliefs you are angry about on other people and you can stop fighting those people over those projected beliefs.
What is particularly striking to me is bundling of two factors here: - Loss of hearing - Identity built around loss of hearing To me these two are distinct. I don't value people based on their disabilities or lack there of. So for me the ability to fix a body's physical deficiency is always a good thing. It makes life better for the person inside the body. These arguments, that I called stupid,