An international team of experts have published consensus-based care recommendations to help clinicians deliver high standards of care to adults with myotonic dystrophy 1.
Work has begun in Professor George Dickson’s lab to refine the gene therapy product.
Sarepta Therapeutics has announced that it will be working with Dr Zarife Sahenk from Nationwide Children’s Hospital in the United States to develop a gene therapy for CMT.
Sarepta Therapeutics has released an update on the four boys dosed in its ongoing gene therapy trial.
We would like to inform you that recruitment for AveXis’ STRIVE-EU gene therapy trial has started at Great Ormond Street Hospital.
Monitoring the progression of muscle-wasting conditions is essential to ensure they can be managed well.
An old drug could help boost new treatments for people with SMA
If you have a progressive condition, finding ways to look after your health to help you stay well is vitally important.
Many of the conditions we support at Muscular Dystrophy UK are rare, but few are as rare as LMNA-CMD.
Professor Jenny Morgan and her team at University College London (UCL) have shown that a type of cell death called necroptosis has an important role in Duchenne muscular dystrophy.