These are exciting times, with fresh news about research developments appearing every week.
Find out about the latest research news below and also visit our Breaking News in Research page.
A new version of CRISPR/Cas9 has been used to remove the toxic RNA that causes myotonic dystrophy.Read more
The US-based company, AveXis, will be initiating a multi-centre trial named STRIVE. This study will be testing their gene therapy, AVXS-101, in babies with SMA type 1.
Two research studies using data from the UK Myotonic Dystrophy Patient Registry have been published recently. The registry is jointly supported by Muscular Dystrophy UK and MDSG.
"This is disappointing but not unprecedented in the regulation process. Another Duchenne drug, Translarna, was initially rejected at an early stage but is now available."
Results from an MDA-supported study show that progressive resistance exercise is safe and can significantly reduce muscle weakness in children with CMT.
We are pleased to announce that we are investing over £650,000 into seven new Duchenne muscular dystrophy research projects that came out of our grant round.
We are investing over £290K into two new Ullrich muscular dystrophy research projects. This was largely made possible thanks to the incredible support of some of our Family Funds.
A recent study has increased understanding of the underlying biology of sporadic inclusion body myositis (sIBM) and identified a potential therapeutic target.
As part of the EU-funded PREFER project, we are helping to organise a focus group for people with myotonic dystrophy and their caregivers.
It was not known whether facioscapulohumeral muscular dystrophy (FSHD) affects bone health, and a recent study published in Muscle & Nerve begins to address this question.
A gene therapy has been shown to be safe and effective in treating mice with limb-girdle muscular dystrophy type 2E (LGMD2E).