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.
Joint letter to NICE regarding the suspension of the appraisal process for ridisplamRead more
Wave Life Sciences have announced the disappointing news that the development of the drug suvodirsen for DMD has been discontinued.
Sarepta’s exon skipping drug, VYONDYS 53™ (golodirsen) has been approved by the Food and Drug Administration (FDA). The FDA regulates drugs in the USA.
Preparatory work for the clinical trial is underway and progress is being made towards the development of next-generation microdystrophins.
Roche Genentech announced the disappointing news in a letter to the Duchenne community.
RCOT has launched a survey to identify the top 10 research priorities for occupational therapy in the UK. More information can be found at the RCOT website: www.rcot.co.uk/otpsp
Senior Research Fellow Dr James Sleigh as the winner of our first Research Image Competition.
MDUK-funded research has shown that a new type of molecular patch could be promising for DM1.
Santhera Pharmaceuticals has announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has designated vamorolone as Promising Innovative Medicine (PIM) f
Facio Therapies has announced the completion of a ‘real-world data’ study monitoring the progression of FSHD and its impact on daily life.
Sarepta Therapeutics have announced new data from its trial testing SRP-9003, a gene therapy for limb girdle muscular dystrophy (LGMD) type 2E.