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Year 2008! A new Start...
Years Resolution.. Determination
A year as come and gone again... And what a year its been! A lot has happened over 2007.
One of the big changes has been the name of the charity - Parent Project United Kingdom! We are now known as Action Duchenne, we didn’t think the name was appropriate anymore because the Duchenne boys thought it was just involving parents so everybody decided a name change would be suitable! Also we decided to have a proper launch and the most appropriate time we felt was at the 5th International conference in London at the Holiday INN between 2nd & 4th November - the conference went very well and was very informative! Professors, researchers and scientists from all over the world working with Duchenne Muscular Dystrophy came and did presentations for the families, explaining the in's and out's of the disease.
Throughout the year Action Duchenne organised fundraising events like love runs, the Manchester Bupa Run, great north run, sky diving and rock climbing! All the events raised very good mounts of money for research, events were also put on where they put on a dinner and had live auctions. Also its not only the adults and Duchenne boys that raised money, even children from schools wanted to help by doing a sponsored walk round the school. the nursery and little ones did 5 laps, the older infants did 10 and the juniors did 20.
Who says the kids of today don't care!
We have had donations from different partners, one organisation called The Professional Footballers Association - donated £500!
Thank you everyone involved with your contribution, every little helps!
Research.. On the front line
Research has come along a lot this year - a treatment called Exon Skipping which is where you get a drug (Morpholino Antisense Oligonucleotides) injected into your muscles which patches over the messed up DNA produces a functional but short protein of Dystrophin - meaning the disease is less severe, alleviating symptoms! Turning Duchenne into a less severe Muscular Dystrophy called Becker with a practical normal life expectancy.
This year on Tuesday 18th December made history when Exon Skipping begun its first human clinical trials - Professor Muntoni and his team injected the first foot muscle of a young person living with Duchenne. This first phase clinical trial aims to test whether Morpholino Antisense Oligonucleotides could become a gene therapy for Duchenne in the near future!
To make this possible, Action Duchenne and our families have lobbied hard and petitioned with the government to win over £2 million pounds to fund this research. Action Duchenne has also funded pre-clinical work to develop this unique approach to fixing the faulty gene. We have also got an online petition going, because funding runs out in 2008 - meaning trials will come to a halt so the petition is created to demand funding from the government, from the lab to the clinic if Exon Skipping shows successful!
Petition website
http://petitions.pm.gov.uk/breakthechain/
We have petitioned about the government funding DMD previously...
The government replied saying “The Government fully accepts that everything possible needs to be done to bring an end to the tragic impact Duchenne Muscular Dystrophy (DMD) has on individuals and their families. There have been significant developments over the last few years, not least in research, and some grounds for hope.” The determination of all those involved - charities, the pharmaceutical industry, and Government - to find a cure for DMD will give us the best possible chance of that hope becoming a reality.
This being said shows that the government is finally listening so it’s our job now to keep the pressure on by lobbying the government and Department of Health to get them to help us fight the good fight! “Time is critical! Money shouldn’t be an option, life is priceless! We deserve to live! We have no other options; I would rather die trying than die without trying!
DMD Registry and new clinical trials
Its time to get Registered. There are two major clinical trials planned in the UK for 2008. The USA drug discovery company PTC are planning a trial for their drug PTC124 in 2008. PTC124 is a drug that hopes to be used to treat young people with Duchenne who have a very specific gene variation. It will target premature stop codons to produce almost a full version of dystrophin in muscle. The MDEX team are also developing another trial that will test to see if the morpholonos can be delivered systemically or throughout the whole body.
Both the MDEX team and PTC have asked to use the DMD Registry to recruit patients to the trials.
We have been working hard to redesign the registration process for the DMD Registry. In partnership with Treat NMD we have now streamlined registration and added new data fields of key clinical information. This information is vital to help to accelerate the process of finding patients for clinical trials. You can now register online for the DMD Registry. Its easy to do and we can give you support.
To register go to: www.dmdregistry.org or call Angela Stringer on 02085569955 for more information.
If you have already registered you need to logon to edit and update your data.
For more information on research please go to the research section of the Action Duchenne website...
Happy New Year
A new years coming! Full of hope, exciting trials will be happening! A change is going to come!
I am very optimistic for the future of Duchenne with all what I’ve heard from the researchers and scientists, all the different ways to treat the condition something must be on the way soon. The main hurdles that we face are funding and regular bodies slowing down research with
the usual paperwork, red tape, Ect. I consider the best way to get around this problem would be to forget the mice/rats and lets get out of the labs and into human and make DMD a special case after all its a Terminal Disease with no hope of survival ... people living with DMD, parents of and of course the Scientists and clinicians must start to adopt the 'can do' attitude to research and trials, I have been hearing 'we must do more work' for years now and nobody getting better! People are dying. Action Duchenne is committed to funding key Duchenne Research projects in 2008. This includes further funding for exon skipping, drug discovery and stem cells. We need £2m to get this research funded and to be able to accelerate clinical trials.
In the meantime clinical trials could be performed in humans by injecting stem cells into individual muscles such as the diaphragm or throat muscles, muscles that will improve quality of life! I for one would take at least being able to eat food properly again a start!
I urge people to keep in regular contact with their MP's and make your voice and opinions heard to the government, in 2008 we need to carry on all the great work we have all done and encourage more people to do the same, together we can all beat the monster that is Duchenne Muscular Dystrophy!
Lets show we mean business...
We would like to wish everyone a very Happy New Year. We would like to thank everyone who has supported Action Duchenne!
Let's hope that 2008 is the year a treatment comes available!!! Here's to 2008!
1 Comments
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- Susan
- January 08th 2008
- 22:20pm
Happy New Year, Carl and CONGRATS your petition has hit 1000!