One in every 5,000 boys is born with Duchenne muscular dystrophy, the most common fatal form of muscular dystrophy. The disease is relentless. It slowly weakens the body’s muscles, deteriorating function of vital organs and ultimately - shortens their life. Although there are medical treatments that may help slow its progression, there is currently no cure.
Defeat Duchenne Canada is the country’s only national charity dedicated to ending Duchenne muscular dystrophy. We have provided leadership in research, advocacy, and support since 1995. We‘ll continue until a cure is found to ensure our boys can live long and active lives.
My two brothers are affected muscular dystrophy. One is 18 years old and another is 10 years old. 18 years old teenage boy is okay to his daily life working. His body is okay but he cannot walk. Without this he has no problem yet. He is a boy of middle class family. Is it possible to recover from muscular dystrophy? If yes, which process we can follow? 😭
Of course it is all great, the disease is terrible and things are finally looking brighter with elevidys. Great information too and the future. Well...there is more and more future for kids with Duchenne so that is really positive. A long way to go, probably a decade or more to solve it for youngsters and a much longer way to deal with older patients, fibrosis etcetc. I could do without the story of Emery. Not because of him, he is okey. But all the others and how they look into the cam...not for me nor most other Europeans but okey. Not too important.
I think SGT-003 uses a capsid that is engineered to be much more effective to target human muscles and in fact far less the liver (unwanted). So there are already capsid being developped out of current vectors and this will only get better, which is very usefull since for one we could reduce the side effects (less and less of target action) and lower the dose needed (smaller dose, better targeted = higher efficacy adn lower sides). Good to see more researchers going into that direction. Also I wonder how research is developping in bypassing the human defense mechanism so we could re-use some vectors and trigger an immune-response! Because with most current microdystrophine (but also micro utrophine and GALGT2) it is one shot and then it can't be re-used because immunesystem responses. Since muscles develop etc current microdystrophine therapies are probably not functional through life but gradually wears off. Either we need to get rid of capsids or we need to modulate the immunesystem. I wonder if research is done on that possibility?
Given the preliminairy conclusions, I would like to add that NAG (N acetyl glucosamine) is in 100% pure form readily available everywhere. A 20 Kg kid should take about 2 grams per day. This translates into monthly costs of about 20-25 dollars. Another thougt of mine is that this is not directed at the underlying problems with Duchenne. It probably helps a lot but NAG would have the same mechanism in any muscle. So we can wonder how people who are losing muscle strength, like elderly, could profit from this substance.
I was like this at his age in the 90s, walked up until highschool no one knew I even had DMD as well 33 years strong, keep him optimistic never tell him he can't, the mind is a strong part in beating our disease 💪 💪
Dr Lawrence Korngut is such a caring and compassionate Dr to his patients, and he knows his stuff!!! He is my Dr and I’ve never seen a Dr who really tries to the best of his ability to help his patients. I am so lucky to have him as a Dr with what I’ve got going on, and I wouldn’t go to any other Dr out there. A VERY smart man!!!!
My son also have a duchenne mascular dystrophy..God willing when you read this my comment i want to talk jessie to my son to give inspiration to my son ...
an amazing video about an amazing family that I am blessed to know. thank you for all your hard work in trying to facilitate a cure. It gives me hope that "one day" this disease could be cured.
Buy Creatine supplements and just keep him moving do not expect the same level of activity, do not let him run during gym it will just make him deteriorate faster.
Hi Derek, We stumbled on his diagnosis by accident. He was a small baby and after completing some bloodwork we noticed the mutation and the high CK levels. The geneticist confirmed Duchenne.
Duchenne has a different path depending on the type you have. So some progress faster than others. Indeed he is 5 years old there and he seems to be doing much better than about 95% of the kids I have ever seen with Duchenne. His calfs do not seem to be enlarged either which seems to indicate that inspite the disease he has not been affected nearly as much as most other of his age. So regardless of is future, he is lucky to be like this I'd say as compared to other kids with Duchenne ( I am no expert though).
My nephew passed away at the age of 15 on friday 12 November 2021 due to this MD Ducheen I seen and watch and observed him very clously 😭😭 bad diseases
This disease is indeed the most devastating.I am from India and i lost my 16 years old brother suffering from DMD😥😥due to respiratory and cardio failure on 29 august this year.we tried our best to save him but we couldn't...💔💔 I can't describe my brother pain in words💔💔it is very painful we miss him so much everyday
Not sure if you'll see this but, Thanks Kayla! I remember you! I'm glad you got to see the video! I hope you are well too! It's been so long. I'm still in my struggle with DMD but I am conquering it and enjoying life.
Well...nothing that will cure it.Deflazacort and prednisone are able to postpone the detoriation for a couple of years. With side effects that differ a bit from person to person. What people could do is go for an alternative route which is by using nutraceuticals that are (pretty) safe to use. We must realise that there are a lot of quite promissing neutraceuticals out there but for some reason, after initial testing most are not followed up by Phase II let alone Phase III trials. So either tey are in vitro or mostly performed on mdx mice. Mice are not humans and there are quite a few examples where the dystrphic mouse got a lot better but humans weren't. It still to my mind is very sad that there is ample of focus on pharmaceuticals which especially remain to be proven safe over so many years and very little on nutraceuticals that are know to be safe and have done well in prelimanairy testing. Having said all that, it seems to be more than reasonable to get kids with Duchenne Resveratrol. Ina very recent study people with either BMD and DMD had significant benefit even while those tested were already in advanced stages (DMD 10 yrs old or more, BMD up to 30 yrs or more). The authors themselves noted that to them you should start using RSV as soon as possible. In this case it was given orally 3 x 500 mg per day. Adverse effects were mild and going back to 1000 mg resolved the side effects which happened on one patient. Other studies suggest 25 mg/kg which translates into (say a 15 Kg child) 375 mg/day. Curcumin is another substance with a lot of promise. Long story short: you should go for a liposomal form (which re readily available) since it is almost not bio-available in its standard form (well: very little is bio available in the standard powder form). Creatine: most studies show a reasonable effect and it seems very safe. Advised dosage: 0,2 gr/Kg. So if the kid weighs 15 Kg it is 3 gram best on an empty stomac in the morning. CoQ10: some studies show effects others not so much. Note that quite a few studies looked for instance nly at the heart but not at, say, the calfs muscles... Omega3 Fish oil: psitive effects on various markers. Seems to be better in this case to go for oil that is higher in DHA than in EPA which is atypical. Green Tea Extract: most studies show improvement. Look up research on this. N acetyl cystein produced incredible effects in mice and (I believe) also in humans. But: at a 0,2% in mouse feed it ALSO had serious adverse effects. This tends to be a high dose. Most tests in mice are at 0,1%. In a study in 2019 it led to strong upregulation of Utrophin and had very significant effects on DMD (MDX) mice. But: this one is tricky and I would not use it without regular checkup with your doctor. Interesting preliminairy results: N-acetyl Glucosamine. Dosage unknown but it stabilises the cellmembrane and leads to a firmer attachment of the sarcolemma resulting in elss tears. First indication is that it works like a shock absorber. But dosageunknown as it is tested as we speak in mice right now. Not glucosamine. It has to be 100% pure NAG (N acetyl d glucosamine). This is a very safe products with little evidence though but it is now tested for Duchenne for a reason. Astaxantin: to me it is mindboggling why no one has tried this on dystrophic muscles. It is a very very potent anti oxidant. It is very safe and has produced significant results in people with various froms of muscle atrophy. It also showed a very significant drop in footballplayers CK levels after excessive training. Ataxanthin stabilises the cellmebrane due to the way it positions itself in the cell. It leads to mitophagy and brings down quite a fewmarkers in those people that are also elevated in Duchenne. In cell lines with even modest amounts of astaxanthin easily obtainable due to a supplement the effects on muscles was striking. No proof at all that it would work in Duchenne but I would try it. Epicatechin: seems to be a bt liek Astaxanthinin its effects, again it was not tested in Duchenne but is test in now in BMD. No muscle stbilsation but it leads to mitophagy and has had very signiicant effects on people with diabetis and sarcopenia. In general with all these things try to get the right dosage out of scientific literature on the subject and get your kid checked by your octor and if you want to go this route inform your doctor. It might be that some of these substances interact with prednisone etc.
totally agree im 24 living with this. I also wish treatment was available in canada. I too live in fear but try to live my life to the fullest. thanks for sharing
