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Dr Pertusati will produce drugs that could potentially improve how energy is used in muscles, to find out if they might be of treatment use for ADSSL1 myopathy.
Dr Pertusati will produce drugs that could potentially improve how energy is used in muscles, to find out if they might be of treatment use for ADSSL1 myopathy.
Dr Pertusati will produce drugs that could potentially improve how energy is used in muscles, to find out if they might be of treatment use for ADSSL1 myopathy.
Dr Nathan Hodson and his PhD student will look at the metabolism of people with Becker muscular dystrophy and examine whether eating more protein will benefit their physical strength and quality of life.
Dr Nathan Hodson and his PhD student will look at the metabolism of people with Becker muscular dystrophy and examine whether eating more protein will benefit their physical strength and quality of life.
Dr Nathan Hodson and his PhD student will look at the metabolism of people with Becker muscular dystrophy and examine whether eating more protein will benefit their physical strength and quality of life.
This project explores changes in mitochondria in the muscles of people living with myotonic dystrophy type 1 (DM1) following 12-weeks of weightlifting training. Understanding what happens to mitochondria in these muscles may help identify therapy goals for treating DM1.
This project explores changes in mitochondria in the muscles of people living with myotonic dystrophy type 1 (DM1) following 12-weeks of weightlifting training. Understanding what happens to mitochondria in these muscles may help identify therapy goals for treating DM1.
This project explores changes in mitochondria in the muscles of people living with myotonic dystrophy type 1 (DM1) following 12-weeks of weightlifting training. Understanding what happens to mitochondria in these muscles may help identify therapy goals for treating DM1.
Professor Thomas Gillingwater and colleagues previously developed gene therapy to treat X-linked spinal muscular atrophy (XL-SMA); however, they were unable to test this due to a lack of animal models. In this project, the researchers will characterise a newly generated mouse model, which is much needed to test new treatments for XL-SMA.
Professor Thomas Gillingwater and colleagues previously developed gene therapy to treat X-linked spinal muscular atrophy (XL-SMA); however, they were unable to test this due to a lack of animal models. In this project, the researchers will characterise a newly generated mouse model, which is much needed to test new treatments for XL-SMA.
Professor Thomas Gillingwater and colleagues previously developed gene therapy to treat X-linked spinal muscular atrophy (XL-SMA); however, they were unable to test this due to a lack of animal models. In this project, the researchers will characterise a newly generated mouse model, which is much needed to test new treatments for XL-SMA.
Dr Stephanie Duguez and her research team aim to develop a blood test that can detect molecules called sphingolipids, the amounts of which are different between people with a muscle-wasting condition and those who do not have a muscle-wasting condition. The hope is that these blood tests can be used to improve the diagnosis of people with neuromuscular conditions like spinal muscular atrophy (SMA), FSHD, inclusion body myositis (IBM) and myasthenia gravis, and measure the progression of their condition.
Dr Stephanie Duguez and her research team aim to develop a blood test that can detect molecules called sphingolipids, the amounts of which are different between people with a muscle-wasting condition and those who do not have a muscle-wasting condition. The hope is that these blood tests can be used to improve the diagnosis of people with neuromuscular conditions like spinal muscular atrophy (SMA), FSHD, inclusion body myositis (IBM) and myasthenia gravis, and measure the progression of their condition.
Dr Stephanie Duguez and her research team aim to develop a blood test that can detect molecules called sphingolipids, the amounts of which are different between people with a muscle-wasting condition and those who do not have a muscle-wasting condition. The hope is that these blood tests can be used to improve the diagnosis of people with neuromuscular conditions like spinal muscular atrophy (SMA), FSHD, inclusion body myositis (IBM) and myasthenia gravis, and measure the progression of their condition.
In this project, Professor Chris Clark from University College London will be using an MRI technique called the Dixon method to measure the progression of Duchenne muscular dystrophy (DMD) by looking at the water and fat content in the muscles of people with DMD.
In this project, Professor Chris Clark from University College London will be using an MRI technique called the Dixon method to measure the progression of Duchenne muscular dystrophy (DMD) by looking at the water and fat content in the muscles of people with DMD.
In this project, Professor Chris Clark from University College London will be using an MRI technique called the Dixon method to measure the progression of Duchenne muscular dystrophy (DMD) by looking at the water and fat content in the muscles of people with DMD.
Professor David Brook and his research team from the University of Nottingham will be investigating how useful a group of antibiotics called macrolides are in reducing symptoms of myotonic dystrophy type 1 (DM1). If successful, the team will set up a clinical trial to test their findings on people with DM1.
Professor David Brook and his research team from the University of Nottingham will be investigating how useful a group of antibiotics called macrolides are in reducing symptoms of myotonic dystrophy type 1 (DM1). If successful, the team will set up a clinical trial to test their findings on people with DM1.
Professor David Brook and his research team from the University of Nottingham will be investigating how useful a group of antibiotics called macrolides are in reducing symptoms of myotonic dystrophy type 1 (DM1). If successful, the team will set up a clinical trial to test their findings on people with DM1.

NICE publishes draft guidance on efgartigimod for treating generalised myasthenia gravis

Orange graphic with 'BREAKING NEWS' written in lime green

NICE recommendations

Although not NICE's final guidance on this medicine, the draft guidance outlines the committee's current recommendations.

These are:

  • Efgartigimod is not recommended, within its marketing authorisation, as an add-on to standard treatment for generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor antibodies.
  • This recommendation is not intended to affect treatment with efgartigimod that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

What does this mean?

If accepted, this means people who are not currently receiving efgartigimod through the NHS in England will be unable to access it. However, people who are already being treated will be able to continue to receive it until their doctor considers it's no long necessary.

The NICE appraisal consultation document can be read in detail here and is now open for consultation.

The NICE committee recognise that clinical trial evidence suggests that efgartigimod plus standard treatment improves symptoms and ability of people to carry out their normal activities in comparison to standard treatment alone. But they felt there was some uncertainty about the extent to which this evidence would be applicable to the people who would receive efgartigimod in the NHS under the usage being appraised.

NICE says their decision is also due to uncertainties in the economic model which makes the likely cost-effectiveness estimates for efgartigimod uncertain. The committee disagreed with some of the cost effectiveness estimates put forward by the company manufacturing efgartigimod and concluded that overall the treatment would not be within what NICE consider to be acceptable cost-effectiveness estimates for use of NHS resources.

Our response and next steps

It's very common for NICE to not recommend a treatment at this stage. The decision often changes after the consultation stage and a second committee meeting has taken place – for efgartigimod, this committee meeting is scheduled for 16 November.

The draft guidance recognises there is a clinical benefit from efgartigimod. Strong evidence was presented to NICE from clinicians and patients about both the impact of generalised myasthenia gravis on people with the condition and on caregivers; and about the positive experience of people receiving the treatment.

MDUK urges all parties to work closely together between now and November to address the challenges raised in the draft guidance and to find a solution that means new patients can benefit from efgartigimod when clinically appropriate.

How you can share your views

We will continue to work with all parties involved and to present the patient voice in partnership with Myaware.

If you are currently receiving efgartigimod and are affected by this news, we are keen to hear from you so we can incorporate your views into our response.

Please do get in touch by emailing our Campaigns teams

Those affected by this announcement can get in touch by ringing 0800 652 6352 between 10am-2pm on Monday to Friday. Alternatively, you can email us on info@musculardystrophyuk.org

SMA Health Inequality Survey

We’ve built a strong partnership with Spinal Muscular Atrophy UK over recent years, collaborating to secure access to SMA treatments and working together as co-secretariat of the UK SMA Newborn Screening Alliance. Our latest piece of joint work sees us coming together to work with Roche Pharmaceuticals on a project exploring health inequalities faced by people affected by SMA in England. Please note, Roche Pharmaceuticals have funded this project through an independent agency and are partnering with SMA UK and MDUK to capture this data. Roche has no access to this data but have had editorial input into the questions in the survey.
We’ve built a strong partnership with Spinal Muscular Atrophy UK over recent years, collaborating to secure access to SMA treatments and working together as co-secretariat of the UK SMA Newborn Screening Alliance. Our latest piece of joint work sees us coming together to work with Roche Pharmaceuticals on a project exploring health inequalities faced by people affected by SMA in England. Please note, Roche Pharmaceuticals have funded this project through an independent agency and are partnering with SMA UK and MDUK to capture this data. Roche has no access to this data but have had editorial input into the questions in the survey.

About this article

Date Published:
1 September 2023

Campaign Autumn 2023

We hope you enjoy reading your autumn edition of Campaign newsletter, written especially for you and keeping you connected with the work you help fund.
We hope you enjoy reading your autumn edition of Campaign newsletter, written especially for you and keeping you connected with the work you help fund.
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