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.
The results from the MDUK-funded research project gives insight into the drugs, ezutromid
The ENMC is an international organisation that supports research for neuromuscular conditions and strives to facilitate communication among scientists and clinicians.
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.
Santhera Pharmaceuticals has announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has designated vamorolone as Promising Innovative Medicine (PIM) f
Muscular Dystrophy UK is investing £1.2 million into 9 new research projects this year, bringing our total research portfolio to over £10m.
Audentes Therapeutics has provided an update on its drugs in development for myotubular myopathy, Duchenne muscular dystrophy, myotonic dystrophy type 1 and Pompe disease.
Capricor Therapeutics has released positive preliminary results from its HOPE-2 Phase II clinical trial testing CAP-1002 in steroid-treated boys and young men with DMD.