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August 22, 2004

Potential Cure for Muscular Dystrophy

This is very good news

Scientists believe they may have found a way to treat muscular dystrophy.

There is currently no cure for the muscle-wasting disease, the most common form of which affects one in every 3,500 children. Most die young. But scientists at the University of California, San Francisco, say they have identified a defect in a key protein which may trigger the disease. Writing in the journal Nature, they said the discovery could lead to new treatments to fight the disease.

Now that would be very good news, wouldn't it? The new research reveals an acetylcholine transporter, which is something they didn't even expect to exist. Acetylcholine is a chemical released by nerve cells to make muscles contract, and like most all signalling chemicals has to be broken down so it won't hang around forever after having completed its job. They used to think it disappeared naturally, being broken down into acetyl and choline for recycling back into more acetylcholine. The new research indicates that there's a protein devoted to breaking it down faster than it normally would, a transport protein, and without this protein it builds up and causes problems. The Amazon poison curare is an acetylcholine blocker, which inhibits your ability to contract any muscles, leading to death, while some nerve agents stop the breakdown of acetylcholine to produce uncontrollable muscle contractions. It's an extremely important chemical.

However, as my friend who works at UCSF says, some "science reporters" know so little about science that what they report is often a rather addled version of what the scientists are trying to explain, so you can get a clearer view by simply going to the source, in this case UCSF's new release. I'd continue blogging on it, but I couldn't write any clearer than the UCSF release, so go give it a quick read. Interesting stuff.

August 22, 2004 in Science | Permalink


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How soon before they make the company sell the medicine below cost to Canada?

Posted by: Walter Wallis at Aug 23, 2004 12:02:44 AM

Great post George...and good news.

Posted by: Stephen at Aug 24, 2004 12:56:37 AM

If they find a cure or a preventative treatment, it will be a miracle, and I for one will be happy for the many lives it will save.

Unfortunately, it will come too late for many who suffer from it now...my wife being one of them.

And I feel so damned helpless.....

Posted by: LC Brendan at Sep 3, 2004 6:26:05 AM

My grandson Sumit, is a handsome, intelligent 9 years old very sweet young kid. He has been diagnoised with Becker Muscular Dystrophy.
My concern is that with the disease the diet is very poor. I hope that doctors must ask about the diet. I do feel tht in many ethnic groups, the female are blamed for carrying the defective gene. How about testing male and his parents? In case of my daughter, her sister, her mother ( myself) and mydaughter are tested. But the boy's father and his parents are not tested. His father has sex problems, having erectile problems. He also has particular limping style like his father
( in this case gradfather). Is it possible to check all factors before blaming the female.
About help, my grandson is denied for wheelchair. I do not know where to go for wheelchair?
In cases, like this, the parents do need counseling as their marriage falls apart. In extended families like my daughter they need more counselling. Please help. I hope that very soon you may find a cure. How about diggging into Ayurvedic study?

Desperate and frustrated in New Jersey.
p.s. My grandson's grandparents are always moaning about his situation and try to get advantage by openly talking about the child's disability to parking attendant, and try to make him more crippled. Please help.

Posted by: Desai Bhanu at Nov 23, 2004 2:40:22 PM

I too am afflicted with MD at the age of 10, now I'm 21 yrs. old and my muscles continue to degenerate. I hope that this real and I'm teerribly hoping for a cure.

Posted by: Marlowe Jason R. Landicho at Nov 28, 2004 11:42:34 AM

Dear Sir/Madam,
I am a Boy (15 Years) Currently Residing in Pakistan. I have some problems with my Mucsles. My Fingers Dont Open Fully and there is no movement is some fingers. They can feel everything but i cant move them with my will. Doctors here say its a Mucsle Weakness. My Feet Also have foot drop. They can't bear the weight of my Body unabling me to Walk freely. by the grace of GOD. I am currently Walking but with the help of "Bilateral AFO's" (Plastic Made Shoes From feet to just below knees)
So if there is a possible cure Avaiable in Science for this kind of disease Kindly Let me know with Some Details.

Posted by: Abdul Ali at Dec 14, 2004 1:02:10 PM

From Georgia, USA

Hello everyone,

I just found out yesterday, that on Saturday my 61 year old mother was diagnosed with MD (Muscular Dystrophy) she went to see a Doctor due to she's been falling more than usual, and she sometimes had difficulty keeping her head up, and at times this would happen while driving. My mother is a very "proud" woman and stubborn to match. And this has taken her life to a 180 degree turn... she was the one who was working in Home Health helping others - now she's the one who being helped... as I mentioned she's very stubborn, and seems to think that she'll be back to normal in a couple of days... treating this like the common cold. It saddens me to hear about this, and the worst thing of all - is that I live over 1000 miles from her. Talk about feeling helpless. Luckily my older sister is there with her, and keeps me posted. But it's very difficult picturing my mother like this, when I know the type of person that she is... and that person is now deteriorating slowly. Physically first, then it will be mentally - unless they truly DO have a cure for this. I pray that they will for my mothers' sake, and many others out there!

God Bless.

Posted by: Viktoria H at Jan 10, 2005 8:42:35 AM

My husband's brother's daughter who is only 7 yrs old is suffering from MD. They are in India. She does go to get physiotheraphy. We want to help her out. We want to bring her here and give some better treatment. Once i read in the newspaper that researchers at the State University of New York at Buffalo are working on Arachnid spider's venom to find a cure. I couldn't find more info on this. Has anyone heard @ this? Its very sad to see her in this condition. She is so smart and pretty. We can only pray to GOD for some miracle to happen.

From VA

Posted by: Vincy George at Jun 17, 2005 1:11:24 PM

Hi Dr.
I'm 47 years, Egyptian. I had MD [(Muscular Dystrophy] my legs got thinn and I no longer walk. then my arms got weak. I found a treatment in Singabour, but too much. I ask you if you have a free treatment for me, I'll be thanks a lot. this desease is very bad,it's taking a guy from eyes.
Said El Banna

Posted by: Said at Jun 30, 2005 7:22:22 AM

My sister Jess is 16 years old and has MD and CP. Can any one cure her? I would give any thing to know what she is "really" like.
Younger sister Kate 14 Years old

Posted by: Kate at Aug 2, 2005 11:14:52 AM

i'm suffering from becker muscular dystrophy past 10 years now my age is 28 . can you suggest me of any medicine to cure . i can walk fast but i have troble to calim stairs, runing and jumping .

Posted by: ajaydas at Aug 4, 2005 3:52:58 AM

Dear Sir,
My mother is suffering from muscular dystrophy which is most probably the limb girdle type of. We live in India and though there are almost all medical facilities here but still there is always an apprehension. It has been over 16 years she started observing the problem and gradually and gradually she has now reached a stage that she might have to take the wheel chair in a year or two. It is extremely painful for me to see her in this shape. There is no solid evidence about the origin of the disease and there has been no one in our ancestral family affected with muscular dystrophy. Could it have been that something went wrong while she was giving birth to her second child before 18 years because it was a Caesarean delivery and I believe that the Nursing home wasn't really an established one? I am eagerly waiting for your response. I am asking this question because before this there was absolutely no problem with her. So is there any possibility that some nerve or cells might have been damaged while the delivery? Please do let me know if what I doubt is possible and if there is any good news regarding the cure. I'll be highly obliged.

Yours Truly,

Posted by: ashu at Aug 24, 2005 11:36:41 AM

Dear sir!!!
My cousin is suffering from Muscular Dystrophy. The Lab reports shows that the quantitiy of Creatin phospho kinase is 1150u/l which should be 190u/l.The amount of Aldolase is 14.1 u/l which should be 8U/L. Please tell me that is there any cure for this disease?

Muhammad Suleman!

Posted by: Muhammad suleman at Oct 23, 2005 12:53:50 PM

I am from pakistan swat. I am a patient of Becker's Muscular dystropy. I want to know weither it curable or not. Kindly infrom me about it.

Posted by: Aftab Ali at May 19, 2006 11:54:56 AM

I was diagnosed with facioscapulohumeral in 1987. I have noticed a great loss of strength in my legs over the last 5 years.I am now 34 years old I will be in a wheelchair soon if no cure is found.I would be a guinea pig for research if needed.

Posted by: Ben at Jun 26, 2006 8:33:53 AM

I have a two years old son . Last week I understood that my son suffers from Muscular dystrophy. I'm looking for a cure for my son and I will do everything for him.While I was searching I understood that there is genetic cure which can help my son beacuse he is so young. Would you please give me a little information about this.

Posted by: Atefe at Jul 25, 2006 12:55:54 AM

My nephew is suffering from muscular dystrophy. We were told that there is no cure available. He had carried out some ayurvedic treatment where they do oil massage etc. But its just temperory relief.

Later i came to know of one ayurvedic doctor who is known to cure all sorts of uncurable disease. We visited him and now he is under his treatment for the last one and a half months. His CKP value has since come down from 19000 to 11000 in 25 days and now to 2000 in the next 25 days.
We feel there is some improvement.
We hope for the best.


Posted by: mani pr at Aug 18, 2006 11:07:33 AM

Dear Sir,

My brother(17 years), suffering from MD. Right now he is not able to walk/move. Its difficult even to lift his hand to have morsel of food. I am glad to know about cure for MD. Please let me know where to contact for curing this disease. I will be very greatful for your kind help.


Posted by: Abdul Salam at Sep 12, 2006 9:36:56 AM


I am 30 years old and effected by Facio Muscular Dystrophy. I know there is no treatment of this, I belong to middle family I want to know how I improve my condition? I am able to walking but face problem to runing,jumping,steers, lifting weight.

Posted by: Nadeem at Sep 20, 2006 4:37:19 PM

hello every one,
my name is vamshidar and i am from india my sister whose age is about fifteen years is affected with muscular dystrophy and her diet is very poor how to improvise her eating habbits and help her in surviving for a long time docs please help us out in this regards might not be cure but atleast a recovery for her life so that her mom can become happy.

Posted by: vamshidar parepally at Oct 3, 2006 8:57:36 AM

I have 5 siblings including myself suffering from LGMD and am looking for researchers who may want to study our family to find a cure for others. Thanks.

Posted by: T.V at Oct 6, 2006 1:44:13 PM

Where is the incentive for a cure? Answer, there is none. The medical establishment is a business, and like any business they need customers.

One signer of the Declaration of Independence (Dr. Benjamin Rush), who was also George Washington's physician predicted the following. "Unless we put medical freedom into The Constitution, the time will come when medicine will organize into an undercover dictatorship."

Posted by: randy at Oct 7, 2006 12:41:50 PM

dear reader:
I have MD LGMD IIB there is this association www.mdausa.org which is continually doing some research about drug testing and so forth, I would like for an expert to put on easy words what are the new methods or treatments that are over there.
PD. If there is any cure, please let me know

Posted by: Roberto Matthews at Oct 16, 2006 3:44:03 AM


I am a patient of Muscular Dystrophy and I was able to walk till the age of 20 and after that I had an accident and fractured my thigh bone. I had to rest for 4 months and since then I am unable to walk. I am 25 right now and still in a better condition than most people of my age. The most important thing is your will power. If your mind feels you can do something, then you can and I know one day me and others like me can walk around freely. Scientists around the world are looking for a cure and the good news is that they have found the cure for mices or rats. The only problem is finding a harmless virus to transfer the cure to human body. They have found a harmless virus but the human immune system kills it as soon as it enters the body. In this case, the human body is an enemy of itself. So, the cure is very near....maybe 2-3 years away. So keep your fingers crossed you guys :-)

As for the people with kids having MD, the best thing you can do for them is to keep them happy. Don't let them feel they have a disease. Also, make sure they dont gain a lot of weight. The more the weight, the more the problems. Make sure they eat healthy food and sit in a good position. You have to take care of the back too..to prevent it from stooping. The best way to do this is to keep the television set at a higher place and ask the child/person to lie on a lower place on his stomach and then watch tv while raising his head and back a bit. This will prevent a lot of problems. Also, stretch their hands, fingers, tendons (part that joins the leg and feet..just above the heal) twice a day. Get them night splints if you can to keep their legs straight and free from contractions. Encourage them to do as much as they can on their own. Don't worry if they fall once in a while..just help them stand on their feet again. I have a serious DMD case, but I played cricket till the age of 20 (cricket is a game like baseball). If I didn't had the fracture..I would have been walking even now. I did have problems in running, climbing stairs, getting up when I fell down......but I didn't mind it one bit. I always kept my spirits up and I am happy as I am right now. My fingers dont open fully..life is very hard.....but I only know to fight the odds in my life..and I will win....and so will you!! Just feel the soul within you......feel the power of your mind!! I completed my Masters in Computer Science and I work online on projects from US, UK, AUSTRALIA, MALAYSIA, etc. I won't let my problem come in the way.....I just find some simple solutions to make my life easier..there is always a way to do things if you think hard. No one helps me with my daily routines (bath, etc.) I do it all on my own. I have a 4-legged stool with some small wheels below each leg and I freely move around. My hands are very weak.....but I just do my best. I just need to think that my hands can help me and they do :-)

If any of you needs some more info., then just drop me a mail at [email protected] If I made a change in any of your lives and you would like to donate some money to me..to help with my treatment or anything, you are welcome :-) I will reply all queries!! I want everyone with DMD or BMD to feel happy about themselves..I am going through it but still I am happy. I hope I inspired some of you to fight your disease. The disease is as big as you think!! Be happy as long as life lasts..dont ruin the life you have by worrying about the future. There are many others with far worse problems.....we are lucky to get so many happy moments and so much love..just treasure them all..live for the moment....To the Health and Well-Being of All.....


Posted by: Vaibhav at Nov 6, 2006 1:30:04 PM

Here is a link for the latest update on Muscular Dystrophy. Just scroll to the bottom of the page to find the status of current research :-)


Posted by: Vaibhav at Nov 6, 2006 1:37:14 PM