CureDuchenne is a national nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy. Duchenne is a progressive muscle-wasting disease that impacts 1 in 3,500 boys. Boys are usually diagnosed before age 5, in a wheelchair by 12 and most don't survive their mid-20s. Currently, there are no approved treatments or cure for Duchenne. CureDuchenne funds promising research to find a cure for Duchenne. For more information, visit www.CureDuchenne.org
My son has DMD but was too old to receive gene therapy.. look at your beautiful boy climbing those stairs!! Our only joy is that our boy is now a man and although he has many aids to help him in his life.. he lived independently (with carers of course) but he is also 40 years old now.. 🎉 he is enjoying life and he fills our family with joy ❤
TYSM for this comment! I'm glad to know they're with you! how are they doing? my brother is 23 and he's good, he graduated from college last year and is now looking for a job
I adopted three brothers out of DSS here in South Carolina. The older two were diagnosed about six months into my foster care. I was told at the time that they would not be able to pass 18 and we’ve just had terrific care here in the upstate of South Carolina. My oldest son is pretty much bedridden and he can still feed himself, but that’s all he will get in his chair some, but it’s very painful. He has some deformities in his legs as a result of the DMD. My second son who’s 24 is doing very well as a Part-time job editing videos here at the house and is doing really well.
My nephew has DMD and he has the worst kind unfortunately, can you PLEASE PLEASE tell me everything about this gene therapy? You’ll save a life . Thank you 🙏
Well that little one has some Large calve muscle now.. Going to have POPEYE legs in a few years.... Hope all improvements are on the steady...Keep up that hard work
What it is rarely about are the costs of these therapies. Would be nice if all these companies were as open about these very important matters too and explain why the costs are that high and how much profit they are going to make. We know what happened to Emflaza in the USA after a patent was bought. We know how Ireland refused to accept Translaran due to costs. Duchenne hits boys all over the world not covered by any insurrance. Nice to have medications that could help in the near future, but how will it help if there are no funds to obtain them. How are all these companies going to deal with that and what do they think it all will cost society. I think what Ireland did with Translarna is not only sort of brave but also good so to show society is not going ot accept rather absurd costs for very little real gain.
No, this is disgraceful and evil! Stop disguising eugenics. They're already aborting more black people than white, they already disallow disabled people to be born, more LGBTQIA are aborted than straight, more females are aborted than males, all advertisements of designer babies are images of white males. There's advertisements of anti aging which has nothing to do with sicknesses or diseases, if it was only about sicknesses and diseases they wouldn't of ever thought of touching the human germline. So don't bother denying this is racism, sexism, ableism, communism, eugenics and capitalism. They do not have the right to mess with a person's genes just because they perceive something about them as a "problem". Autism is not a problem, society claims it values all humans equally, since society wants to rub us out of existence you clearly do not see us as equal, these are full of discriminatory thoughts like Hitler doing eugenics. The problem is clearly their view of autism and other primarily genetic conditions. I love being autistic, I wouldn't give it up for the world. Molecular biologist Miroslav Radman writes, "Mutagenesis has traditionally been viewed as an unavoidable consequence of imperfections in the process of DNA replication and repair. But if diversity is essential to survival, and if mutagenesis is required to generate such diversity, perhaps mutagenesis has been positively selected for throughout evolution." Do you really want to bring us to extinction? Evelyn Fox Keller explains: "We now know that mechanisms for enduring genetic stability are a product of evolution. Yet a surprising number of mutations in which at least some of these mechanisms are disabled have been found in bacteria living under natural conditions. Why do these mutants persist? Is it possible that they provide some selective advantage to the population as a whole? Might the persistence of some mutator genes in a population enhance the adaptability of that population? Apparently so. New mathematical models of bacterial populations in variable environments confirm that, under such conditions, selection favors the fixation of some mutator alleles and furthermore, that their presence accelerates the pace of evolution." The mutants behind autism and other conditions like Down Syndrome offer some great advantages to the human race, diminishing the genes is a great risk because without those mechanisms there is no asurety of genetic stability pushing us in the direction of extinction, and delay the adaptation process. Psychologist Howard Gardner warns: "With the coming of age of genetics, the danger magnifies. Beyond doubt we will discover genes that are important for reading alphabetical scripts; and there is already evidence that a small set of genes may be related to reading problems. As with the brain evidence, such information can be helpful for early intervention; but it could easily be used for stigmatising purposes. Indeed, it might become relevant for marriage prospects, holding a job, securing insurance, or even eugenic purposes. And no doubt, especially in our interventionist society, individuals with a genetic predisposition for reading problems will look into different kinds of genetic engineering or therapy. It is possible that such interventions will work and have no negative side effects, but it is perhaps more likely that they will have unanticipated effects. And we might even want to consider which valued human abilities - eg. spatial or pattern recognition skills - might be placed at risk were we to target our interventions specifically at reading disorders." Do you really want to destroy all alternative perceptions and ways of thinking? Don't you know how many abilities you are going to destroy and how impoverished you are going to make our world because of your cultural myopia? Each time they have tried playing God they have only caused harm. Who caused the climate change? Scientists playing God trying to control nature, did these Gods anticipate the climate change? You are not only messing with humans, this whole earth is interconnected, you are messing with the entire ecosystem, with all life. How many species have been brought to extinction because of humans manipulating nature? there's endangered species today thanks to humans manipulating nature. If we fail to understand and take care of the natural world, it can cause a breakdown of these systems and come back to haunt us in ways we know little about. A critical example is a developing model of infectious disease that shows that most epidemics - AIDS, Ebola, West Nile, SARS, Lyme disease and hundreds more that have occurred over the last several decades - don’t just happen. They are a result of things people do to nature. The diseases you claim you want to cure were caused by doing this, so why are you doing it again? Was the world ready for COVID-19 to strike? I doubt it. World War II was caused by eugenics, why are you following Adolf Hitler's steps? Mutations are not random or accidental, malaria is endemic in Africa and Africans have developed mutations that protect them from malaria through adaptation, the sickle cell mutation is a defence against malaria, Europeans don't have these mutations, if a European goes to Africa they are more likely to get a disease, what would of happened to Africans if you eliminated that defence mechanism? The Africans would of been eliminated. It was mutations that enabled the Europeans to survive the 14th century bubonic plague. Editing one gene may cure a disease but at the same time make them more susceptible to other diseases. Do you really believe mutations that enable survival are really just accidental and random as these assume? Again, this is wicked and pure evil to think we don't deserve to be born just because we are different. CRISPR-Cas9 is a direct violation of human rights, especially human autonomy. They are full of discriminatory thoughts and don't you touch us without our consent!
Very good, but what I am missing in all these things are answers about the future. Nothing definitive but I guess most parents would like to know when thngs could potentially come to market if Phase III studies work out fine.
All this amounts to a couple of extra years where kids, treated in time, can walk. So this disease even with givinostat is devastating. But we are getting somewhere out of the predisone/prednisone era where side effects are so bad you can wonder if you want to give your kid these medications. I wonder where Vamorolone+Givinostat will get us. Or with upcoming treatments that are in phase three, like pamrevlumab that focusses on fibrosis and other treatments. Like some that do take over the function of Dystrophin. May be the combination of these will get people really on the way.
I am the mother of a 14 years old boy with DMD who is no longer ambulant for years and I am very eagerly waiting for the Vamorolone/Givinostat combo, hoping it could hopefully limit the damage caused by the disease.
Finding therapies is one part of the story making these affordable for patients is another . What are Pharma companies doing to address the patient access
