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Funding medical research for Duchenne Muscular Dystrophy

Posted by CureDuchenne on Thursday, 14 December 2006 14:38:42

Hi David

Last year you joined the All Party Muscular Dystrophy Group to support our Charity's campaign (Parent Project UK) to win new Government funding for research to find a cure for Duchenne Muscular Dystrophy (DMD). As you know DMD is a severe muscle wasting disease that leaves our sons without any muscle and often dead by the time they are 20. There is currently no cure. The exciting thing for our families is that the UK is leading the world in new gene therapy research - exon skipping - that could provide the first real treatment for this terrible disease, as well as other diseases such as Cystic Fibrosis. The problem is that the researchers need around another £20m to get this gene therapy to the market. After a meeting earlier this month, the DoH have stalled our funding with the offer of a round -table talk.

When individuals in the City are receiving £20m bonuses, we think that its time for our society to get its priorities right and fund translational medical research for dying children?

Will you continue to support our campaign as a member of the All Party Group Muscular Dystrophy and ensure that the Government properly funds this exciting new gene therapy project ?

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Posted by Boz4J on Sunday, 17 December 2006 00:48:47

http://www.i117.photobucket.com/albums/o62/bozena_03/147_4794_1.jpg

Posted by Boz4J on Sunday, 17 December 2006 00:54:34

Is not possible to open this picture from the blog but all who are intrested to see it , please go to that website .This is my dedication for all especially for you MR.Cameron.Once again , please help our kids.

Posted by mwb72 on Sunday, 17 December 2006 14:03:37

My son is 7yrs old and has DMD. It is heartbreaking knowing that his condition will deteriorate more so than it is now. Please help to ring fence the money needed to fund the research and create the treatments that will help to save our childrens lives.

Posted by kozmicstu on Sunday, 17 December 2006 15:45:00

Have you all been diverted here from another website, perchance? I'm amazed at the somewhat disproportionate amount of suffering there is on this website...

I wish you all the best and join you in hoping a cure is found.

Stu

Posted by Boz4J on Sunday, 17 December 2006 17:28:51

Thank you kozmic Stu for you comment , is heart worming I hope that we will have more people seeing our soffering.I hope that more and more PEOPLE WILL SUPPORT OUR FICHT TO CHANGE IT AND TO FACE IT AND TO FIND THE CURE..Our kids can not fight them selfes in the Parlaments of the whole world.Parents are so tired about dealing with sympthomes of the doisease that just small of group of them have enough strengh to lobby for the research.Because those kids are dying in the ypoung age , they never become famouse to have big inpact on public life.Our kids need help from healthy adult world,otherewaise ying closed in small quaiet badrooms will continuie, in accompaniaments of mothers tiears.IT TIME TO STOP THAT DISEASE.Prommise to me --all you who will ever read this post that you all will help accordingly to your possibilities.Thank you.

Posted by fdirosa on Sunday, 17 December 2006 21:08:30

Duchenne Muscular dystrophy is a rare disease that is in the forefront for rare genetic disorder research efforts by many research institutions. There has been many discoveries in the past year alone that can break the "doom & gloom" prognosis given to our young boys. If we want to begin to make a difference in the prognosis of rare diseases we need to start treating/curing them rather than putting them on the "back burner" because of their rarity. The studies occuring may not only treat duchenne MD but many other rare and common genetic disorders. Now the the diseases this research can potentially treat is not rare but common. We are on the verge of great treatments for many incurable diseases, now is the time to push forward even harder. We need to invest more now than ever before into this research, not less.

Posted by bensdad2 on Sunday, 17 December 2006 21:13:04

Come on David - my son is nearly 6 and has Duchenne. He is such a delight to have, but as others have said, the future is bleak for him and many others. It is only money that stands in our way., Please help Ben, and others like him.

Posted by mc69thriller on Sunday, 17 December 2006 21:44:29

Come on anyone who can help us out!!!

Money shouldn't be an option not for peoples lifes, government should be able to
fund us, for that cure we so rightly deserve, instead of buying ugly expensive statues, lets make
me and all the people with Duchene Muscular Dystrophy better!

20 million is nothing to the government, even 30m is nothing to them, anyway even if it was its for a
good reason, it will be worth it, it would show that our government has a heart!

Please stop this heartache, lets change history!

Come on its Christmas!

Posted by RaceAgainstTime on Sunday, 17 December 2006 21:56:56

I am 33 years old and have Duchenne Muscular Dystrophy, it has left me unable to move, confined to a wheelchair and dependant upon a ventilator to breathe. Not only does this terrible disease have a devastating effect upon those affected but on whole families who often go without sleep and such luxuries as holidays in order to look after their disabled child. My elder brother passed away from DMD 6 years ago. No sibling should have to go through losing a sibling, no parent should ever go through losing a child . It breaks my parents hearts to know that they face losing another son unless genetic research like exon skipping can be transformed into a viable treatment and funding is desperately needed in order for this to happen. All we are asking for is £20m which is just 0.026% of the total NHS budget and the sooner a treatment becomes a reality the more millions it will save the NHS in the long term. With your support we can make a difference together!

Thank you,
Mark
www.race-against-time.co.uk

Posted by babalu on Sunday, 17 December 2006 22:42:27

hi, we have friends whose son was diagnosed with DMD.We think that is very important to have more fundig for the research. It seams like there is money for everything else but not enough to save our kids.

Posted by andysdad on Sunday, 17 December 2006 22:47:56

David,
I have a son aged 8 years old. He and his sisters do not know yet the terrible future that faces him but some day soon we will have to tell him and the girls. The exon skipping project is one major project that can save him and the government must fund it. The evidence is clearly there and we must push the medical ethics bodies to allow us to fast track the trials in boys.
The funding will not be money wasted as it will create a treatment for boys. Pharmaceutical companies will take it on from there. Please act now and have £20m ringfenced for this - it is very very urgent. The charity Parent Project UK is at the forefront of this campaign and can give full information on exactly how this funding will be used.

Posted by tearyeyedmom on Sunday, 17 December 2006 23:56:16

I'm a mother with a 2 year old son with Duchenne Muscular Dystrophy. As a parent I'm asking you to
please fund this critical exon skipping research. The research being done now, may be the miracle we have all been waiting for. It's too late for the many that have died a cruel death from this disease, but you can make a difference in the future, by spending money for a cause that will help many children that will die otherwise. Paying money for statues and memorials is great ,they have their place ( the pigeons really seem to appreciate them). The money that will be funded for this research will have a far greater impact, an impact that will have no boundaries. Please reach out to help our boys with Duchenne and those with other diseases that will also benefit from Exon Skipping.
Thank you

Posted by Boz4J on Monday, 18 December 2006 00:21:23

MR.CAMERON our kids need your help . Our kids need advocates like you and others .We need paople who will start to talk about Duchenne and other mascular distrophies .WE- as a hiuman kind sent people to the Mars .We spent bilions of dollars just to upgrate the plains.we pay milions of dollars for models and actors and sport players...We have houses for milions , we have hotels for thousands a day , we have cars for 200 000 dollars.We buy art for milions .This is fine, let happy people spend money on what ever they like.But the world has to know that we can do more good with the money.The world should know that there are kids waiting on the dead row , waiting for the CURE in the midle of most developed countries .In the midle of richest countries on the world.Riches in the finantial capitals but also in Science.But science needs money to grow.We have money to grow electronics and technology because it pays then ,after it sells.But I think that the World is enough rich to afford some expences for something what maybe will not paid of financially , but only in the SMAIL of thausands kids and parents.Thank you , if you will decide to help us and our kids.Thank you in the name of thausand , thausand young people for being a good ADVOCATE for them.Maybe some day you will become a Prime Minister .Please never forget about Duchenne kids and others.THE WORLD CAN AFFORD TO FIND THE CURE FOR ALL, JUST WE PEOPLE HAVE TO HELP IT TO HAPPEN.Sincerly .Bozena , mother of the only son with Duchenne.

Posted by Boz4J on Monday, 18 December 2006 03:44:02

Woops!We did not send people to the Mars yet.I meant the Moon .Maybe Mars will be next.

Posted by Doogie on Monday, 18 December 2006 13:58:08

We are just an ordinary family, like every other... I have 4 children, all have their ups and downs... just life really.
Until 2 years and 9 months ago that is.... when out of the blue a blood test on my youngest son, 5 at the time, revealed 'a problem'. More tests and heart stopping waiting, and my worst nightmares were confirmed.
My little boy has Duchenne Muscular Dystrophy.

He now has this future to face... we all do. My other children, aged 11, and twins of 10, are trying to come to terms with the brutal facts that their brother is facing a short life, and his ability to run, jump, walk, smile, breathe, its all limited, and every day, every month that goes by, we have to watch him slowly losing the ability to do these things he loves, the things that make him smile... even his ability to smile, that too is going to be cruelly taken.

It is like my little boy is skipping in quick sand, and he is sinking, and there is nothing I can do to help him. We who love him have to watch him slowly sink, slowly suffocate to this awful condition.

PLEASE... you have the power to lift him out of this, the hope for a treatment is so real, so close. I cant pull him out of this without you, without your support. Time is running out.

Pauline Brace, mum to Joshua Dovey.