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Professor Matthew Wood at Oxford University is developing a molecular patch that could be a potential treatment for people with myotonic dystrophy type 1. This research will also help to further enhance molecular patch technology, which will be beneficial for the neuromuscular field in the long-term. (October 2019) Read an update on this project here: Molecular patches show promise in a mouse model of myotonic dystrophy type 1
Professor Matthew Wood at Oxford University is developing a molecular patch that could be a potential treatment for people with myotonic dystrophy type 1. This research will also help to further enhance molecular patch technology, which will be beneficial for the neuromuscular field in the long-term. (October 2019) Read an update on this project here: Molecular patches show promise in a mouse model of myotonic dystrophy type 1
Professor Matthew Wood at Oxford University is developing a molecular patch that could be a potential treatment for people with myotonic dystrophy type 1. This research will also help to further enhance molecular patch technology, which will be beneficial for the neuromuscular field in the long-term. (October 2019) Read an update on this project here: Molecular patches show promise in a mouse model of myotonic dystrophy type 1

Response to 5 News' report on Changing Places toilets

Submitted by Anonymous (not verified) on Tue, 10/29/2019 - 00:00

Clare Lucas, Head of Policy and Campaigns at Muscular Dystrophy UK, which co-chairs the Changing Places Consortium, said:

“It’s incredibly disappointing that almost a third of NHS Trusts have not applied for Government funding to install Changing Places toilets. It is crucial that hospitals have fully accessible Changing Places toilets so that people can attend important appointments or visit friends and family.

Professor Matthew Wood from the MDUK Oxford Neuromuscular Centre is developing molecular patches for spinal muscular atrophy (SMA) that can cross from the blood into the central nervous system. This research could lead to potential treatments that are safer and less invasive than Spinraza.
Professor Matthew Wood from the MDUK Oxford Neuromuscular Centre is developing molecular patches for spinal muscular atrophy (SMA) that can cross from the blood into the central nervous system. This research could lead to potential treatments that are safer and less invasive than Spinraza.
Professor Matthew Wood from the MDUK Oxford Neuromuscular Centre is developing molecular patches for spinal muscular atrophy (SMA) that can cross from the blood into the central nervous system. This research could lead to potential treatments that are safer and less invasive than Spinraza.
Professor Matthew Wood and his team at the MDUK Oxford Neuromuscular Centre are investigating new delivery methods for gene therapy. Although this project will focus on gene therapy for Duchenne muscular dystrophy, its findings will be valuable for the development of gene therapies for other muscle-wasting conditions.
Professor Matthew Wood and his team at the MDUK Oxford Neuromuscular Centre are investigating new delivery methods for gene therapy. Although this project will focus on gene therapy for Duchenne muscular dystrophy, its findings will be valuable for the development of gene therapies for other muscle-wasting conditions.
Professor Matthew Wood and his team at the MDUK Oxford Neuromuscular Centre are investigating new delivery methods for gene therapy. Although this project will focus on gene therapy for Duchenne muscular dystrophy, its findings will be valuable for the development of gene therapies for other muscle-wasting conditions.
Professor Matthew Wood and his team are developing molecular patches with the ability to target the heart and prevent deterioration of heart function in mouse models of Duchenne muscular dystrophy. The aim of this study is to see what further improvements this treatment has on other processes in the heart such as metabolism, and to also test how effective these molecular patches are at restoring dystrophin levels in human heart cells.
Professor Matthew Wood and his team are developing molecular patches with the ability to target the heart and prevent deterioration of heart function in mouse models of Duchenne muscular dystrophy. The aim of this study is to see what further improvements this treatment has on other processes in the heart such as metabolism, and to also test how effective these molecular patches are at restoring dystrophin levels in human heart cells.
Professor Matthew Wood and his team are developing molecular patches with the ability to target the heart and prevent deterioration of heart function in mouse models of Duchenne muscular dystrophy. The aim of this study is to see what further improvements this treatment has on other processes in the heart such as metabolism, and to also test how effective these molecular patches are at restoring dystrophin levels in human heart cells.

Update on vamorolone for treatment of Duchenne muscular dystrophy

Santhera Pharmaceuticals has announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has designated vamorolone as Promising Innovative Medicine (PIM) for the treatment of Duchenne. This is the first of several steps in the Early Access to Medicine Scheme (EAMS), which aims to give people living with debilitating conditions access to medicines that are not yet licensed.

Vamorolone is a steroid-like drug that is thought to have fewer side effects than current steroid treatments for Duchenne. A placebo-controlled phase 2b trial comparing vamorolone to prednisolone is currently recruiting in the UK.

You can read the press release from Santhera Pharmaceuticals here.

 

Previous news related to this story:

11 October 2019

Duchenne trial open for recruitment in UK

A phase 2b trial testing vamorolone is now recruiting at six UK sites (Newcastle, Glasgow, Liverpool, Leeds, Birmingham and London). Vamorolone is a steroid-like drug that has fewer side effects than current steroid treatments for Duchenne muscular dystrophy. More information about the trial and contacts at each trial site can be found on clinicaltrials.gov

For more information, read this press release from the VISION-DMD research group. 

28 August 2019

Results from vamorolone trial published

Results from a six-month study testing vamolorone in boys with Duchenne have been published in the academic journal, Neurology. Vamorolone is an anti-inflammatory drug that could be an alternative to steroids. The study showed that vamorolone was safe and improved muscle function in a dose-dependent manner.

Vamorolone is currently being tested in a global phase 2b trial called VISION-DMD, which has UK sites including London, Newcastle, Liverpool, Glasgow and Leeds (Birmingham is not yet open for recruitment). Eligibility criteria and contact details for UK sites can be found on clinicaltrials.gov

7 September 2016 

Duchenne steroid alternative being trialled in the UK

ReveraGen BioPharma has today announced that enrollment has begun for its phase 2a trial testing vamorolone (VBP15) in boys with Duchenne muscular dystrophy. Vamorolone is an anti-inflammatory drug that is similar to steroids but may have fewer side effects.

Trial participants must be steroid-naïve (no current or previous treatment with steroids) and aged between 4-6 years old. The UK arm of the trial will take place at the Newcastle-upon-Tyne Hospitals NHS Trust.

For more information about the trial, please visit http://vision-dmd.info/2a-trial-information/ or read the company’s press release.

 

15 March 2016

Million pound boost for Duchenne research

The European Commission awarded The John Walton Muscular Dystrophy Research Centre at Newcastle University £4.6 million for a new clinical trial to test the anti-inflammatory drug, vamorolone for the treatment of Duchenne muscular dystrophy.

For more information, read Newcastle University’s press release.

To find out more about the latest research into DMD, please contact our Research Line at research@musculardystrophyuk.org or call 020 7803 4813.

 

Duchenne muscular dystrophy
Research

Uniting Britain’s CMT warriors

Muscular Dystrophy UK’s support inspired Peter Neville to cook up a plan which has united and helped over a thousand Charcot Marie Tooth (CMT) ‘warriors’.
Muscular Dystrophy UK’s support inspired Peter Neville to cook up a plan which has united and helped over a thousand Charcot Marie Tooth (CMT) ‘warriors’.

About this article

Date Published:
17 October 2019

Abbie Can

Emily and Anthony Garbutt have set up Abbie Can Family Fund for their daughter, Abbie, who was diagnosed with Facioscapulohumeral muscular dystrophy in September 2018 at the age of 7.
Emily and Anthony Garbutt have set up Abbie Can Family Fund for their daughter, Abbie, who was diagnosed with Facioscapulohumeral muscular dystrophy in September 2018 at the age of 7.
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