Muscular Dystrophy UK is investing £1.2 million into 9 new research projects this year, bringing our total research portfolio to over £10m.Read more
Researchers involved in the OPTIMISTIC trial have published a scientific paper that identifies genetic factors that influence the severity of myotonic dystrophy type 1.
A new study has shown that editing a ‘modifier’ gene can prevent and reverse symptoms in a mouse model of congenital muscular dystrophy type 1A.
Our Research Communications Manager, Dr Jenny Sharpe, recently attended the twelfth meeting of the International Myotonic Dystrophy Consortium (IDMC) in Gothenburg, Sweden.
The FSH Society International Research Congress was held in France, on 19-20 June 2019
The registry is once again live. If you haven’t yet signed up to it, you can do so now, and if you have signed up for it previously, you can go in and update your records.
Positive results found from blocking myostatin in a mouse model of OPMD. This demonstrates that treatments not targeting the genetic cause could still be beneficial.
The US FDA has granted approval to Zolgensma, the first gene therapy for SMA. Although the approval process in Europe is separate to the US, it’s a major step forward.
Santhera Pharmaceuticals announced that it has started a collaboration with the University of Basel to develop a gene therapy for LAMA2-deficient congenital muscular dystrophy.
MDUK-funded researchers have corrected a common mutation causing Ullrich congenital muscular dystrophy in the laboratory.
Sarepta Therapeutics announced today that it has signed an agreement with Nationwide Children’s Hospital (Ohio, USA) to develop a gene therapy for LGMD type 2A.