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 research study using data from the UK myotonic dystrophy patient registry has been published. The registry is funded by MDUK and the MDSG.
The US Food and Drug Administration has granted Orphan Drug Designation to MYO-101, for the treatment of limb girdle muscular dystrophy 2E.
Acceleron Pharma has announced the US Food and Drug Administration has granted Fast Track Designation to ACE-083.
The conference included talks and posters on a range of neuromuscular conditions. We were delighted to see lots of MDUK-funded researchers in attendance.
Researchers in Scotland have found that a protein called neurochondrin may play a role in spinal muscular atrophy (SMA).
Benitec Biopharma releases update on its OPMD gene therapy programme
Summit Therapeutics has announced it has completed dosing participants in its Phase II PhaseOut DMD trial.
Solid Biosciences has announced that the FDA has allowed the high-dose group to be included in the IGNITE DMD trial.
Pfizer has announced the first participant for its US-based gene therapy trial, was recently dosed at Duke University Medical Centre.
New research has shown that certain mutations in a gene called SCN4A are associated with some cases of ‘cot death’, or sudden infant death syndrome (SIDS).