New Treatment for Duchenne Muscular Dystrophy Patients | Cedars-Sinai
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- Опубліковано 14 лип 2024
- A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles-including the heart muscle-in #DuchenneMuscularDystrophy patients, a new study published in the international peer-reviewed journal The Lancet shows.
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These are trial results?
Meaning its not FDA approved?
(Like we can trust the FDA since big pharma funds them.)
Assuming both answers to be yes...
What type of stem cells are being used to do the infusion?
Why are they only helpful to improve upper body muscles and not lower body muscles?
What is the difference?
Is the difference in the type of stem cell?
Where its administered?
When its administered?
What is the answer?
If we can find the type of stem cells being used, we don't need to wait for the FDA to fuck around while people deteriorate daily.
Just because this country is in the grip of big pharma doesn't mean other countries are.
I give zero fucks about patents.
Only about my young friend. I'm prepared to do whatever it takes. He doesn't have 5 years to wait for FDA approval. He's not legally old enough to make these kind of decisions, but I know what his answer would be if he had any idea of what is coming. He would shout, DO IT!
I'm sick to death of reading about all of these promising treatments "in the pipe", that languish in FDA trials while they figure out ways to make these methods into treatments rather than cures. Its money before people's suffering and it pisses me off.
Did you look at nutraceuticals you can try, that are very safe and could potentially help since science to various degrees is either promissing or in fact got results. I can empathise with you and the fact that you give zero about these trials and won't go into them as to why they are needed (how about your kid dying because of an immune response?).
Things you should look up and then decide to use or not are:
Resveratrol: got signficant results in 3 x 500 mg daily dosing in an open label study recently. This mostly older patient group with either Duchenne or Becker got better over time, not worse. 3 x 500 mg got no serious side effects but of note is that research indicates 25/50 mg per bodyweight is a working does. So if a kid weight 15 Kg that is 300-600 mg/day or 3 *100 or 3 x 200 mg. Best thing is lipsomal in which case I would lower the consumption to 100 mg/day. Otherwise the dosage I mentioned and take it with fat.
NAG (N acetyl glucosamine): very cheap, very safe. I s abundant in mothersmilk. Long story short: in duchenne mice it stabilses the sarcolemma. This means it works at the basis of duchenne in these mice. Resveratrol does that too in part, by strongly upregulating utrophin which is very similar to dystrophin, Since it is found in mothers milk I would also take it with fat. Of note is that in these Duchenne mice the force in the muscles went up by no less than 50%! I have never seen anything similar in any research paper and believe me: I read loads of them. But humans are not mice and many very promissing drugs/nutraceuticals have failed in the translation to humans.
Omega3 fatty acids: SO DHA and EPA. Total fatty acids 3 gram/day. It works such that various markers. Doesn't taste well, so get one that is made with some fruit flavour and that has been manufactured such that fishy taste and burps are eliminated. I personally would buy it like such a fluid and put both resveratrol powder and N acetylglucosamine (NOT just GLUCOSAMINE!) in it and dissolve it. NAG tastes sweet so it won't be a problem if they are pure they sould dissolve and leave the oil whitish. If they are full of fillers probably not....
Curcumin: like most of the above we do know very well how this one works and also the effects in duchenne mice was clear. Difficult to obtain usefull levels in humans, unless you use micelles (in pills) or liposomes (in fluid). The latter is an easy way to get it where it should need. Curcumin seems very safe.
Taurine: some of the best results ever were doen with N acetyl cysteine. When we look at how good it worked in mice and how for instance utrophine was expressed all around all cellmembranes it seems the gold standard. However, when the dose is bumped to twice the amount found in researchj there were considderable and negative side effects. Now in the US, this is considered a drug and can be bought over the counter so that is bad too. However: more recent research has found that most if not all of these effects are due to taurine which is made in body of NAC. Taurine only in very high doses has similar side effects. It is ove rthe counter, cheap and very promissing and used by many people in high doses for a long time. So i would still be cautious and go for 250 mg * 3 which seems to be very safe.
Creatine: 200 mg/kg bodyweight, So if a kid weighs 20 kilo that means 4 grams/day. Most work indicates it has positive effects on muscle strength and activity levels. I would never go beyond 5 grams per day btw.
GTE: consists of a few substances that are known to work well and are in fact currently researched by pharmiacists. Especially epigallocatechin on one hand and epicatechin on the other. Epicatechin is already tested in people with sarcopenia and the results were pretty striking. Also in people with diabetis. Microscopes show it has a very significant effect on mitochondria, the powerhouses of cells. Due to duchennes these mitochondria get destroyed and become dysfuntional. Epicatechin can correct it. So this one is after the cellmembrane ahs ruptured and Ca2+ enters the cell destroying the mitochondria. Epicatechin is quite expensive and difficult to get in a 90% pure powdered form. You should aim for 50-100 mg day in adults. Which seems to be like about 1-2 mg/kg. So in kids about 30-40 mg would suffice. Pahramcists used (-)-epicatechin first (natural form) and got these results. They are now going for the (+)-epicatechin which cannot be found in nature but is supposed to be 400x stronger (and I fear about as expensive). However: you can also buy Lindt Chocolate 70% pure. In various tests, this chocolate which uses a different proces and thereby retains the epicatechins, it shows to have 30 mg/40 gram of chocolate. That comes down to about a dollar per day may be less. The powdered form is 25 gram for 65 dollar. So that is about 0,10 ct/day. A lot cheaper.
COQ10: 200 mg day. Various results most positive. More muscle strength, protection of the heart muscle etc. Go for a liposomal form I'd say end get down to 50 mg. I have no proof of this, but liposomes work longer and get levels in blood up by 8-20 fold.
There a bunch more. In this case if I had to chose between these I would go for resveratrol, dissolve it in Omega3 fatty acid and mix it in yoghurt or mild. I would surely add taurine and Creatine. Bot have a good safety profile, evidence is stronger too and they actin different ways.
I would however for sure notify my doctor to check my son, most of all if he is on prednisone,prednisolone or deflazacort. I have to say that I understand your desparation. If you are willing to gamble the life of a young boy by getting him drugs that have not been tested for safety, the above are a piece of cake. But I would not let that desparation get the best of me and go to a doctor to at least check him up regularly.....Anyways: may be this helps?
Please help me with the treatment of the muscular dystrophy?
Please help i have DMD, i'm 36 years old and still going strong. When will this treatment be available, life has been extremely difficult. I'd appreciate it! God bless!
How long have you been suffering this..??
@@khushi_sharmaa ye problem birth k sath aati h but iske symptoms 6...7 sal ki age se dikhne lgte h slowly slowly ye disease bdhti rhti h unfortunately I am suffering this disease and ye 5..6 trha ki hoti h
@@Rockstar-vh4jg apne koi treatment krwaya?
You have what treatment do
@@aruna250 no treatment available of DM😭😭
is there any way to receive that treatment right now? i have very late stage DMD and that would probably help significantly if I can get that, i don't know what age group can receive the treatment but I am 30 and i am really at the worst stage and it's a very difficult way to live, i would do anything for any kind of improvement
Can you talk to me about DMD ..
I can tell you something about DMD
murdock university in western australia have some medication will be able to cure 30% of patience. god bless u
@@andypham7884 is it only available in Australia ?
@ayesha rasheed Please don't give me any advice about the treatment?
27, I’m sorry to read that 😢 it’s such a cruel disease 😢
Im Muscular Dystrophy Patient My Heart Skips a beat every minute 💔🤒
Is there any treatment now??
My muscles were fine earlier but gradually they have become weak. Doctors here say there is no cure I am living a very sad life
my son affected DMD please help me
Please help im from sri lanka
Help me
I have DMD 30 years old still going strong. Is the treatment safe??
Hi My Son Nithun 7 Years Old He is sitting and standing very slow i am consider may doctors he is tolled DNA test ,now Result is came my son hex on value 47 to 52 is deletion is there . now conform DMD Is there .now Is the treatment is safe?
Go to Dr. Ananda Stira, pastor in Tiruchirapalli, Tamil nadu. [Ministry of Jesus ] that person healed many Muscular destrophy kids through his God Jesus Christ.
Stem cell therapy is how much useful for MD patients and how to avail. If someone from Pakistan. Plz guide
MD ki koi treatment nhi hai world wide mjhy tu yahi btaya hai neurologist ne
@@muhammadharmain7 please give me your no as my son is also DMD patient
Hello sir plz help sir my two son dmd patients I want come to your country for the treatment of my sons age of son is 11 an second son age is 8 years from Pakistan
It's really difficult to approach them. Did you contact with another doctor for DM treatment?
Please help my son
Plz give email address tq
My son too😔😔😔😔😔
Please help me my son
Hello sir Me muscular dystrophy patient please reply to me
I am Bangladeshi, please help my son
Plz give email address tq
I'm from Bangladesh..plz help my brother
Hello how is your son? My brother passed away. My son has this illness. What city you live in.
Invent new medicine after all of them passed away affected by DMD ducenne muscular dystrophy
Nee treatment?? Nothing at all.