Audentes Therapeutics has provided an update on its ASPIRO gene therapy trial. The study is evaluating AT132 in young children with X-linked myotubular myopathy.
Researchers at University College London (UCL) have developed artificial ‘mini-muscles’ that could speed up testing potential treatments for muscle-wasting conditions.
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.