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The second phase of an LGMD 2B study is looking for participantsRead more
Sarepta Therapeutics have announced new data from its trial testing SRP-9003, a gene therapy for limb girdle muscular dystrophy (LGMD) type 2E.
Pharmaceutical company, Catabasis, and American charity Jain Foundation have announced a preclinical research collaboration to study edasalonexent in dysferlinopathy (LGMD 2B).
Muscular Dystrophy UK is investing £1.2 million into 9 new research projects this year, bringing our total research portfolio to over £10m.
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.
The 12th UK Neuromuscular Translational Research Conference was held in Newcastle on 4-5 April 2019.
One adult and six children with LGMD2D were treated with the gene therapy MYO-102. The treatment was safely delivered to their muscles and a new clinical trial is being planned.
Sarepta Therapeutics has announced preliminary results from the MYO-101 trial.
MYO-102, a gene therapy for limb girdle muscular dystrophy type 2D has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA).
Myonexus Therapeutics has initiated the first-ever gene therapy (MYO-101) trial for people with limb girdle muscular dystrophy type 2E (LGMD 2E).
Myonexus Therapeutics gave an update on its LGMD gene therapy programmes at Sarepta’s recent R&D day.