Investing in the UK’s research and development sector is essential for people living with a muscle wasting and weakening condition to benefit from access to life-changing treatments.
We’re calling on the next Government to maintain the UK’s position as a world leading centre for research excellence.
The UK has a valued reputation as a research centre in developing life-changing and life-extending therapies and treatments. Today, research into muscle wasting and weakening conditions is at a promising stage, with a current pipeline for new treatments that was unthinkable just decades ago.
“We set up The NorthStar Network nearly 20 years ago to improve standards of care for boys with Duchenne muscular dystrophy (DMD). Today, the improved standards have helped many boys extend their lives into adulthood. MDUK support has helped us to establish standardised outcome measures for treatments and therapies, driving faster more effective research.” Professor Francesco Muntoni, Paediatric Neurologist at Great Ormond Street Hospital, and Chief Investigator for NorthStar
Despite the great advances that have been made in recent years, there are still challenges in attracting the best staff, expanding clinical trial capacity and reforming the drug approval system. It’s therefore essential that the next Government continues to invest and support investment into the UK’s research sector, especially as early access to treatment is vital to securing better health outcomes for our community.
Acting quickly is critical for administering many treatments for people living with muscle wasting and weakening conditions. We know of cases when delays, sometimes of months or even years, make the difference between someone accessing a drug or not. Delays to making potentially groundbreaking life improving treatments available can be devastating for patients and their families.
People with muscle wasting and weakening conditions urgently need new treatments. That’s why we fund pioneering research to find new and effective treatments so that more people can benefit today, and we can transform the lives of future generations.
“Although the specialists can’t say for certain how much Translarna has helped Luca’s condition, we think it’s working.”
“He’s not too stiff and he’s got good mobility and flexibility. He does everything everyone else does at school. Luca saw his friends riding their bikes and that encouraged him to learn. At first, he didn’t have the muscle power. But he kept pushing and persisting and then he just got it. He found the freedom of riding a bicycle.” Pedro Fernandes, dad to Luca who lives with Duchenne muscular dystrophy
But we can’t achieve the best outcomes alone, we rely on the Government to help support charity-funded early-career researchers. This support has helped us grow our grant portfolio, and importantly, this funding also helps train the next generation of researchers and scientists in the field of muscle wasting and weakening conditions.
We lead the drive to get faster access to treatments for families in the UK, because we know streamlining the process for treatment approval so it is faster and more efficient is among our community’s top priorities for healthcare. The Government also has a vital role to play in this work. Sometimes, a drug may only be made available by regulators in certain parts of the UK. Sometimes, a patient may lose access to a drug because the clinical trial they were on has ended. The Government needs to find new and innovative ways to better the regulatory environment to prevent these issues from stopping UK families accessing treatments.
“As an adult with SMA, I’ve been on Risdiplam for three years and it has improved my life in every aspect. I now have the stamina and energy to work four days a week and take part in social activities at the weekend. It has given me a chance to do things that previously I would not have had the energy to do!” Lauren, who lives with spinal muscular atrophy.
We’re calling on the next Government to deliver five key priorities for our community. One of these priorities is maintaining the UK’s position as a world leading centre for research excellence.
“It’s vital our country maintains its world-leading research excellence, whichever party wins the next general election. This will enable investment and involvement in international collaborations. As an academic group leader, I continue to benefit from funding from Muscular Dystrophy UK. For me, this funding enables the bench science required as a first step for the development of potential gene therapies for Duchenne and Becker muscular dystrophy, and also in the training of the next generation of research scientists.” Professor Linda Popplewell.
The next Parliament will be a critical time for research and development into muscle wasting and weakening conditions. We need the incoming Government to take the above steps to help unlock more research breakthroughs, increase equitable access to new treatments and ensure our community can benefit from these treatments as early as possible.
