This month has been a time for real reflection for millions of people across the UK, The Commonwealth and the entire world. The passing of Queen Elizabeth II sent shockwaves across the world. Her Majesty has left a void which many are still feeling days and weeks afterwards, and will continue to feel. For me personally, her loss was a signal to truly appreciate the senior figures who have played such an important role in my life. It has made me closer to my family, and has sparked me to fully embrace our new adventure in home-schooling for my daughter. If you would like to talk your feelings through with one of our Support Officers, please do not hesitate to get in touch with info@actionduchenne.org or call 07535 498506. We’d be happy to help you, you are not alone.
Clinical trials in Duchenne
When Action Duchenne was founded in 2001 there were no Duchenne clinical trials taking place in the UK. Since then the number of clinical trials and research into Duchenne has grown exponentially. From the hundreds of Duchenne families we support, we hear that although some families are still unable to access clinical trials, many families are able to enrol in clinical trials if they wish to. Some are even in a position to need to choose between multiple studies.
There are many reasons families choose to take part in clinical trials. They aid in the discovery and development of new drugs to treat Duchenne, allowing doctors and scientists to understand more about the condition and how to treat it. When considering a clinical trial, it’s important to remember that not every clinical trial is successful and some potential treatments may not be better than current treatments. However, all research is valuable, aiding in the discovery of better treatments now and in the future.
If you are, like many families we support, starting to look into clinical trials, a great place to start is the ClinicalTrials.gov website. This database lists all the privately and publicly funded clinical studies conducted around the world. Although it is a mine of information, please don’t feel you need to understand all the complicated jargon and wording. That’s where we come in.
Action Duchenne 2022 International Conference – support and information sessions
Here at Action Duchenne we have a number of ways to support you through your decision. You can dial in to our online pre-Conference explainer event on Saturday 22nd October. This Zoom event is open to all and will give you an overview of the basics of Duchenne research and clinical trials. The online event is particularly helpful for our newly diagnosed families who will be joining the conference for the first time.
Our aim is that once you have attended the online event, you will then have the knowledge and understanding to confidently attend our International Conference on November 11 and 12 in Leicestershire. At the Conference, our international expert speakers will take you on a deeper dive into more specific technologies, such as gene therapy, steroid replacements, stem cell therapy and emerging treatments.
The international Duchenne community is stronger together. After all this time we are proud to give everyone the opportunity to share experiences, knowledge and power at this unmissable event.
In order to make the most of both the online then the in-person sessions, I recommend you speak with your clinician to find out your specific genetic mutation. You will then be able to identify which clinical trials you could potentially look into, either by checking the ClinicalTrial.gov website or by asking your clinician to help you find the right trial for your family. This is also a great opportunity to let your clinician know you would like to be considered for clinical trials.
We can also help you with this. You can contact us if you have any questions or worries, or perhaps it’s just not making sense to you. It is very common for parents and families to feel overwhelmed by the ‘science’ behind Duchenne, so you are not alone. We came to 20 locations over the Summer, bringing Duchenne science to the community, and although we did an incredible job of helping families understand, we’ve only just scratched the surface and have hundreds more families to reach. We’ll be back out on the road next year, bringing science to life, so please look out for a venue near you.
The psychology of a diagnosis
One of the highlights of this month has been reading Dr Jo’s inspirational piece about finding hope after a diagnosis. The part which resonated most strongly with me was the ‘worry controller’, deciding when you will think about things, and when you can put things to one side. When a Duchenne parent or carer gets support from one of Action Duchenne’s Support Officers, they are able to create a safe space, whether it is a phone call, meeting or video call. The safe space can be their time to tackle their Duchenne worries, really explore their feelings, then ‘put them to bed’ to get on with life.
Celebrating Professor Muntoni
Earlier this month, we joined the global Duchenne community in celebrating the recipient of the World Duchenne Organization Leadership Award, Professor Francesco Muntoni. This award is the highest recognition for individual achievement, following Professor Muntoni’s ground-breaking career spanning four decades. His leadership has had a profound impact on advancing therapy development for people living with Duchenne. A heartfelt congratulations to Professor Muntoni, for this well-deserved award and thank you for his hard work and dedication to the Duchenne community.
Further funding from Pears
I am delighted to share the fantastic news that, thanks to funding from Pears Foundation, we have, once again, been awarded a grant from disability charity Contact. Previously Pears Foundation enabled our Santa’s Grotto 2020 and peer-to-peer support work over lock-down. This time, Pears will provide a small grant that will allow us to fact-find the support needs for children transitioning from primary to secondary education. This is one of the most stressful events in a young person’s life. Many young people living with Duchenne start to see their mobility decline around this time, often they find they are using their wheelchair more, or losing the ability to walk altogether. Many struggle with isolation in their teenage years, and find the transition to secondary tough.
We are delighted to receive this much-needed grant from Pears Foundation via Contact and look forward to sharing our findings with you over the next few months.
And finally…
I’m pleased to share with you some photos from our first weeks home-schooling Annie, my daughter. It’s going to take some time to adjust to the new arrangement, but will settle down eventually.
Keep safe and I am looking forward to seeing many of you in November at the Conference.
Florence
London Marathon 2022
