Helping us understand congenital myopathies: a new study of changes to an important muscle protein is providing insights

Some congenital myopathies and other muscle conditions are caused by changes in the gene that makes a protein called RyR1. In people living with these conditions, different types of changes, known as modifications, have been found on a protein involved in muscle contraction, called myosin. This research, partly funded by Muscular Dystrophy UK, may help to understand RyR1-related muscle wasting conditions and could lead to the development of new treatments.
Some congenital myopathies and other muscle conditions are caused by changes in the gene that makes a protein called RyR1. In people living with these conditions, different types of changes, known as modifications, have been found on a protein involved in muscle contraction, called myosin. This research, partly funded by Muscular Dystrophy UK, may help to understand RyR1-related muscle wasting conditions and could lead to the development of new treatments.

Running the London Marathon for nephew living with muscular dystrophy

In May 2023, Vicky Hartley’s life changed when she was diagnosed with breast cancer and underwent a mastectomy. Fast forward ten months and the 55-year-old paediatric nurse is training to take part in the London Marathon on Sunday 21 April, inspired by her 23-year-old nephew, Oli, who lives with muscular dystrophy.
In May 2023, Vicky Hartley’s life changed when she was diagnosed with breast cancer and underwent a mastectomy. Fast forward ten months and the 55-year-old paediatric nurse is training to take part in the London Marathon on Sunday 21 April, inspired by her 23-year-old nephew, Oli, who lives with muscular dystrophy.

Approval of Duvyzat (givinostat) for Duchenne muscular dystrophy in the USA

On Thursday, 21 March 2024, the oral drug for the treatment of Duchenne muscular dystrophy (DMD), Duvyzat (also known as givinostat), was approved by the FDA (U.S. Food and Drug Administration) for use in boys aged 6 and older in the United States. Duvyzat is the first non-steroidal drug approved to treat people with all genetic variations of DMD.
On Thursday, 21 March 2024, the oral drug for the treatment of Duchenne muscular dystrophy (DMD), Duvyzat (also known as givinostat), was approved by the FDA (U.S. Food and Drug Administration) for use in boys aged 6 and older in the United States. Duvyzat is the first non-steroidal drug approved to treat people with all genetic variations of DMD.

ADHD and muscular dystrophy means your brain and body are constantly fighting each other

Six-year-old Rory has Beckers muscular dystrophy and is neurodivergent. This Neurodiversity awareness week, Rory’s mum Kerry opens up about why having ADHD creates extra challenges with a muscle wasting condition. We also hear about the need for more research into the link between some muscular dystrophies and neurodivergence.
Six-year-old Rory has Beckers muscular dystrophy and is neurodivergent. This Neurodiversity awareness week, Rory’s mum Kerry opens up about why having ADHD creates extra challenges with a muscle wasting condition. We also hear about the need for more research into the link between some muscular dystrophies and neurodivergence.

Our President, Gabby Logan running her first half marathon in 20 years

Sports anchor, podcaster and author, Gabby Logan MBE last ran a half marathon two decades ago, but on Sunday 7 April, she is taking on the London Landmarks Half Marathon in support of Muscular Dystrophy UK. Gabby has been our President since 2018 and has shown her support in various ways, including hosting events and helping to raise awareness of muscular dystrophy, but this will be her first running challenge for us. She has been inspired by watching others taking on challenges.
Sports anchor, podcaster and author, Gabby Logan MBE last ran a half marathon two decades ago, but on Sunday 7 April, she is taking on the London Landmarks Half Marathon in support of Muscular Dystrophy UK. Gabby has been our President since 2018 and has shown her support in various ways, including hosting events and helping to raise awareness of muscular dystrophy, but this will be her first running challenge for us. She has been inspired by watching others taking on challenges.

MDA clinical and scientific conference 2024: Andrea’s update

Muscular Dystrophy Association (MDA) conference took place in Orlando, Florida between 2 and 6 March 2024. Our Research Communications Officer shares her thoughts and the latest developments in the research field of muscular dystrophy.
Muscular Dystrophy Association (MDA) conference took place in Orlando, Florida between 2 and 6 March 2024. Our Research Communications Officer shares her thoughts and the latest developments in the research field of muscular dystrophy.

The role of periostin in Duchenne muscular dystrophy  

The research work of Professor Linda Popplewell and Dr Alberto Malerba reveals high levels of periostin − a protein involved in the formation of scar tissue − in mice with Duchenne muscular dystrophy (DMD). These findings could potentially have a big effect on the development of targeted therapies for people living with DMD.  
The research work of Professor Linda Popplewell and Dr Alberto Malerba reveals high levels of periostin − a protein involved in the formation of scar tissue − in mice with Duchenne muscular dystrophy (DMD). These findings could potentially have a big effect on the development of targeted therapies for people living with DMD.  

Being a mum is the most rewarding experience of my life

Naomi found out she had muscular dystrophy while she was pregnant. Her two sons, Frankie and Freddie, also have the condition. This Mother’s Day, Naomi shares her incredible story about her family’s journey and what being a mum means to her.
Naomi found out she had muscular dystrophy while she was pregnant. Her two sons, Frankie and Freddie, also have the condition. This Mother’s Day, Naomi shares her incredible story about her family’s journey and what being a mum means to her.

Debbie's RHS Chelsea Flower Show Update

Debbie Hoods, Head of Philanthropy and Partnerships at the charity, is leading the work on our garden at the RHS Chelsea Flower Show. Here she explains more about the garden and how preparations are going.
Debbie Hoods, Head of Philanthropy and Partnerships at the charity, is leading the work on our garden at the RHS Chelsea Flower Show. Here she explains more about the garden and how preparations are going.

What are the key takeaways from the Government’s Spring Budget?

On Wednesday 6 March, the Chancellor of the Exchequer Rt Hon Jeremy Hunt MP delivered the Spring Budget, which provides an update on the Government’s economic plans. As this may be the last time the Chancellor makes a budget announcement before the next general election, the measures outlined will likely set the direction the UK Government will take during 2024.
On Wednesday 6 March, the Chancellor of the Exchequer Rt Hon Jeremy Hunt MP delivered the Spring Budget, which provides an update on the Government’s economic plans. As this may be the last time the Chancellor makes a budget announcement before the next general election, the measures outlined will likely set the direction the UK Government will take during 2024.
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