UPDATE (18/09/18): The researchers are still looking for volunteers for this study and have also announced they are recruiting for a biomarker study - read more about that study here.

We would like to inform you of a new clinical trial at Cambridge University Hospital for individuals with mitochondrial disease.

This study will investigate whether a type of B vitamin, Nicotinamide Riboside, is beneficial for individuals with mitochondrial disease. Preclinical research showed that the vitamin increased the number of mitochondria in mouse models, which led to an improvement in symptoms.

To take part, you need to be between 18 and 70 years old and have a diagnosis of progressive external ophthalmoplegia (PEO) and exercise intolerance/fatigue, caused by a single mitochondrial DNA mutation. Individuals who have mitochondrial disease caused by a specific mitochondrial DNA mutation (m.3243A>G) will also be eligible to take part.

If you would like further information or are interested in taking part, please contact Sarah Bird (sc921@medschl.cam.ac.uk ) or Zoe McIntyre (zm276@medschl.cam.ac.uk ) or call 01223331506.

Further Information

View the study information sheet

Latest news on mitochondrial disease

Today (March 8) the Government have announced new measures to ensure the safety of wheelchair users when accessing wheelchair spaces on buses. The announcement follows the Supreme Court case brought by Doug Paulley last year on the ‘reasonable adjustments’ which bus operators should provide for wheelchair users.

Muscular Dystrophy UK Trailblazers are delighted by the announcement as in our End of the Line report (2016) we discovered nearly two thirds of respondents had experienced problems getting on a bus owing to the attitude or behaviour of the driver or fellow passengers. According to the Government news story announcing the new measures, disabled people are “make 10 times as many journeys by bus as by rail”. This makes it even more imperative for there to be ease of access to these services.

Along with the announcement today, Transport Minister Nusrat Ghani has said:

“Passengers with disabilities must have the same opportunities to travel as other members of society, and it is essential that the services they rely on are accessible and work for them.”

Trailblazers manager Lauren West said:

“We are really pleased the Government is taking action to make it easier for wheelchair users to access buses. In our End of the Line report, launched in 2016, we called on the Government to ensure accessibility in transport works so spontaneous travel is possible. With disabled people more likely to use bus travel, the Government needs to ensure disabled travelers have equal and consistent access to buses. We are hopeful these new measures will make this a reality.”

For further information contact Trailblazers: 020 7803 4846 or email on trailblazers@musculardystrophyuk.org

Saturday 17 March sees the return of the Muscular Dystrophy UK Championship after ‘snowmageddon’ put paid to the last Premiership round of fixtures. Jack Bosanquet has taken the time to put together a preview of the weekend ahead and to tell us about why he enjoys playing powerchair football.

My name is Jack and I am 18 years old. I play in goal for Nottingham Forest Powerchair Football Club. I really enjoy powerchair football as it allows to meet different people and it gives me the ability to take part in sport competitively (which I am otherwise unable to do). I really enjoy taking part in a fast paced, exciting sport and the camaraderie that comes from playing in a team.

 

The moment of the season for me so far is when I helped our team to earn 0-0 draw against the then joint league leaders Sevenoaks PFC. This may sound slightly boring but keeping a clean sheet against a good side when playing in goal feels like a real achievement to me. Another highlight was when the captain for our team scored a hat trick in an exciting 3-2 win against WBA Dudley PFC. This was particularly special as the season before he’d only scored 1 goal!

 

Our ambition as a team is to finish in the top 4 this season and, although we are currently 6th, I feel that, if we put in the performances we are capable of in upcoming fixtures, we still have the ability to achieve this goal.

 

In an ideal world I hope to get 12 points from a possible 12 at the upcoming weekend in the league, but as long as we play as a team and perform at our best I will be happy. Personally, I hope to keep at least a couple of clean sheets as well.

 

The fixture I am most looking forward to is the one against the East Riding Electric Eels who we lost 2-1 to, having led 1-0 at half time, in our previous league match. This was an unlucky loss as we played very well, but I think that this time we have a chance of winning because there is a bit of a score to settle!

We would like to invite you to take part in a study about anxiety in Duchenne muscular dystrophy. The study aims to get a better understanding of how to improve support for people living with Duchenne.

Everybody feels worried or anxious from time to time, but sometimes individuals and families living with Duchenne can have these feelings more often than others. Researchers at University College London (UCL) are holding focus groups to explore the experiences of people living with Duchenne, and how feelings of worry or anxiety might affect them. The researchers will also assess whether current methods for measuring anxiety are accurate in people with Duchenne.

The study will be held at UCL and is recruiting individuals with Duchenne (7-18 years old) and parents of children with Duchenne. To participate, you will need to be able to travel to UCL to take part in a confidential group session (1-2 hours long), which will consist of a number of focussed activities and discussions. Group sessions will be separate for parents and children.

Travel expenses will be reimbursed and all participants will receive a voucher as a thank you.

If you would like further information or are interested in this study please contact Dr Kate Maresh (k.matesh@ucl.ac.uk) or Rachel Trimmer (rachel.trimmer.16@ucl.ac.uk) or call 020 7905 2151.

Further Information

Learn about our mental health matters programme

Read the latest Duchenne news

Last week Muscular Dystrophy UK joined forces with NHS England, expert health professionals, and other charities to highlight and address the key issues facing the delivery of neuromuscular services in England.

Our Chief Executive Robert Meadowcroft (pictured) chaired a number of the sessions at the National Neurology Advisory Group (NNAG) Neuromuscular Disease Workshop, which took place on Friday 23^rd February at the University College London (UCL) Institute of Neurology.

Neuromuscular networks were identified as a vital means of improving care through promoting collaboration between clinicians, people with muscle-wasting conditions and their families, and charities such as Muscular Dystrophy UK.

Muscular Dystrophy UK has helped to set up networks across the UK, including the South West Neuromuscular Operational Delivery Network (SWNODN) at Bristol Hospital, which in 2016 was formally recognised as a Network of Excellence for patients with neuromuscular conditions.

In addition to this, Muscular Dystrophy UK has helped secure £5.2 million of NHS funding over the past five years to support people living with muscle-wasting conditions across the UK. This has helped fund a range of posts including new care advisors, physiotherapists, and specialist nurses.

We are also working with neuromuscular networks and groups in Scotland, Northern Ireland, and Wales to ensure there is increased investment in neuromuscular services.

Bobby Ancil, Senior Neuromuscular Outreach Manager, said:

The NNAG Neuromuscular Disease Workshop is just one example of how we are working with healthcare professionals to promote best-practice care for people with neuromuscular conditions.

Our regional neuromuscular networks also ensure that NHS decision makers are aware of the gaps in neuromuscular care and how they can improve the support available to people with these conditions, while saving the NHS money.

If you would like any more information on our work to strengthen neuromuscular services in your area please get in touch with our campaigns team: campaigns@musculardystrophyuk.org or on 0207 803 4826.

On Wednesday Muscular Dystrophy UK Trailblazers held the second meeting of the Young Disabled Person’s Working Group on Employment at the Guildhall. The Working Group is part of MDUK’s policy work on disabled employment funded by the City Bridge Trust. The meeting was attended a variety of young disabled people who are both in work and looking for work which made for great discussion and gave people a chance to share tips with each other on how to navigate the daunting world of work.

Everyone who attended was passionate about removing the barriers disabled people face when trying to find work and very keen to help MDUK create will-informed policy to do this. The disability employment gap has been the topic of several news stories in the past few months, including on the Government’s Strategy to get 1 million more disabled people into work Government’s Strategy to get 1 million more disabled people into work in the next 5 years, which gave the Working Group great deal to discuss.

The meeting was also attended by several members of the City Bridge Trust who were keen to hear about the experiences of young disabled people. James Lee who was in attendance said:

"It was incredibly valuable to hear first- hand from young disabled people about the barriers to employment which they face.  The meeting provided plenty of discussion points, actions to take forward and served as a powerful reminder of how far society still needs to go to ensure equality of opportunity.  Trailblazers provides such an important platform for young disabled people to have their voice heard and the City Bridge Trust is proud to support the valuable work of Trailblazers and Muscular Dystrophy UK."

Clive Gilbert, a Working Group member who has attended both meetings said:

“Another really insightful and well-informed discussion. While securing employment as a disabled person can sometimes feel like an uphill struggle, it seems clear that many of the challenges arise from negative attitudes and unwanted assumptions on the part of employers. Every meeting of our working group brings ample evidence that dispelling these myths would yield immense benefits, not just for disabled people, but also for employers and society as a whole.”

MDUK’s Employability Officer Emma Vogelmann who led the meeting said:

“I am so pleased with how the second meeting of the Working Group went. We had returning members and people who had never been to a Trailblazers event before attend. All of the experiences shared will really help MDUK create effective policy recommendations.”

If you’re a young disabled person and would like to join the working group, email our campaigns team: campaigns@musculardystrophyuk.org 

We attended a celebration event to mark one year of the Department of Work and Pensions (DWP) disability sector champions scheme. The event was opened by the Secretary of State for Work and Pensions, Esther McVey MP and the Minister of State for Disabled People, Health and Work, Sarah Newton MP who spoke of their great pride in the scheme and all it has achieved so far.

Improving access makes good commercial sense as disabled people have a combined spending power of £80 billion. We heard from 9 of the current sector champions about what they have been doing over the last year. It was also announced that three new sector champions had been recruited for insurance, trains and arts and culture.

Below are some of the key points from the event:

• A number of the champions spoke of the importance of disability awareness training for staff so that they can understand the needs of people with different disabilities.
• Attitude is everything are using their charter of best practice to improve access at music venues and festivals.
• England’s Inclusive Tourism Action Group, have created 10 top tips for inclusive tourism.
• There has been an increase in disability visibility within advertising, such as the Maltesers adverts.
• Blackpool have developed an accessible model for bus transportation that they hope other places will adopt.
• A disability toolkit has been created for retailers to help them make shopping centres accessible to all.

You can find out who the different sector champions are on the DWP website.

Parents of children with muscular dystrophy and adults with the condition shared powerful testimonies at the first session of the new All Party Parliamentary Group (APPG) for Muscular Dystrophy inquiry into access to psychological support yesterday.

What happened at the meeting

The session, chaired by Mary Glindon MP, heard from:

• Sue Barnley, whose son Harry has Duchenne muscular dystrophy
• Louisa Hill, whose son Archie has Duchenne muscular dystrophy
• Steve Ledbrook, who has Becker muscular dystrophy
• Jaspal Mann, whose son Kirath has Duchenne muscular dystrophy
• Sarah Mitchell-Innes, who has congenital myotonic dystrophy
• Mathy Selvakumaran, who has congenital myopathy

We are very grateful to everyone on the panel and those in the audience for providing such open and emotive views and experiences throughout the session.

The APPG was keen to establish strong patient perspective themes to take forward during the inquiry. These included:

• needing access to psychological support where there is an understanding of muscular dystrophy and the progressive nature of the condition
• lack of psychological support available alongside support from other neuromuscular health professionals
• recognition that psychological support is required for both people with the condition and also family members
• psychological support should be available close to home as the clinic setting is not always appropriate
• referral processes take many months
• people are forced to turn to private psychological support because of long waiting times or inadequate support

The next session on Wednesday 21st March will focus on evidence from health professionals and professional bodies.

How you can get involved

The APPG would like to hear from people with muscle-wasting conditions and family members about their experiences of accessing psychological support.

There are some questions in the Have Your Say document, and you may wish to answer some or all of these, or alternatively you can email a statement of no more than 250 words about psychological support. Please email your answers to the questions or your statement to Jonathan Kingsley at j.kingsley@musculardystrophyuk.org by Friday 27th April 2018.