The newsletter for our supporters.
Welcome
I’m delighted to welcome you to this year’s spring edition, showing how your support helps us be there for people living with muscle wasting conditions. I was pleased to meet the winners of our President Awards. It’s always such a pleasure for me to be involved with the awards and learn all about the incredible nominees.
Shelia Hawkins, our winner of the Richard Attenborough Award for Outstanding Achievement, shares her story about another way she’s chosen to support our work through a gift in her Will.
You can read our latest research round-up, which includes Dr Daniel Moore’s fascinating work with ‘mini muscles’ and another MDUK-funded project that explores how the brains of people living with Duchenne and Becker muscular dystrophies are affected by their condition.
Thank you for your support – it makes our work possible.
Gabby Logan MBE
President, Muscular Dystrophy UK
President Award’s recognition
Our annual President’s Awards are one of the highlights of our year. They celebrate people in the muscle wasting and weakening community who have gone above and beyond in their contribution to the lives of people living with a condition.
All this year’s nominees were exceptional and clearly determined to do all they can to make a real difference.
The winners’ ceremony took place last month and was hosted by our President, Gabby Logan MBE, and sponsored by Airnow Technology.
The awards recognise achievements in the six categories of research, caring, volunteering, community engagement, fundraising, and an outstanding achievement award.
Research News
We’re making great progress. But there is still so much that needs to be done. Donate today.
Potential gene therapy identified by growing ‘mini muscles’ in a dish
Dr Daniel Moore recently completed his PhD research which focused on identifying a potential gene therapy for a group of muscular dystrophies called laminopathies, using ‘mini muscles’ created in the lab.
These ‘mini muscle’ models behave like the muscles in people affected by these conditions.
Laminopathies are diseases caused by genetic changes in a gene called LMNA, which makes proteins called nuclear lamins.
These proteins play a vital role in supporting the control centre of the cell – the nucleus. In people affected by these conditions, changes to the shape of the nucleus within muscle cells is seen.
This eventually leads to muscle cell death over time.
What happens in the brain in Duchenne and Becker muscular dystrophies?
This project is part of the Brain Involvement in Dystrophinopathies (BIND) study which has been partially-funded through one of our research grants. The aim of the study is to improve the understanding and measurement of dystrophin in the brain.
Dystrophin is a protein which plays a key role in maintaining muscle strength. People with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) produce less or no dystrophin, which results in damage to their muscles.
While there has been a lot of research looking at how dystrophin affects the muscles, there has been less research looking at its impact on the brain, and what happens in the brains of people living with DMD and BMD.
A helping hand
Kim has a titin-related myopathy and lives in Glasgow. She shares her struggles with getting the care she needs, and how our team have been there to support her.
“One of the most traumatic experiences for me has been having my care cut. In 2022, I was assessed as needing 52 hours of care a week, but in July last year I was told at very short notice this would soon be cut to 31 hours a week.”
Our helpline team supported Kim – liasing with social workers and helped her understand her contribution to her care.
Your support enables our helpline team to provide information and advice about all aspects of living with a muscle wasting or weakening condition. If you, or someone you know, needs help call 0800 652 6352 or email info @musculardystrophyuk.org.
NICE recommends Vamorolone to treat Duchenne
When Mandy’s son, TJ, was diagnosed with Duchenne muscular dystrophy at age three, life changed for them as a family. Mandy says, “I went from being a mum of three to also being a full-time carer, trying to hold myself together whilst going through the worst heartbreak I could ever imagine.”
When TJ was five years old, he received the drug Vamorolone for six years via a clinical trial. Mandy believes he gained huge benefits on the drug.
“TJ did amazingly on Vamorolone. At age 11, he was still walking and only needed to use his wheelchair for long distances. His medicalteam would often comment on how great he was doing for someone his age with Duchenne.”
Every day is vital when you’re living with a muscle wasting condition and it is only with your support that we can continue to campaign on behalf of our community.
See the six treatments we’re currently campaigning for access to.
Get involved
Spring Shop
Be one of the first to check out our spring selection of water bottles, gifts, bags and so much more.
Give as you live
If you shop online, you can raise free funds for us while you shop! Simply join Give As You Live (GAYL) to start raising free donations for us, at no cost to yourself.
Lottery
Help change the future for muscle wasting conditions for children like Carmela (above) – as well as have a chance to win fantastic cash prizes – by entering our Weekly Lottery!