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.
MDUK-funded researchers have corrected a common mutation causing Ullrich congenital muscular dystrophy in the laboratory.Read more
Facio Therapies has shown that one of its lead compounds can reduce the levels of DUX4 in a mouse model.
The US based charity, Neuromuscular Disease Foundation, has been awarded a challenge grant to support gene therapy research for individuals with GNE myopathy.
AveXis has announced it is seeking marketing approval for its gene therapy, AVXS-101, in Europe, United States and Japan for infants with SMA type 1.
Work has begun in Professor George Dickson’s lab to refine the gene therapy product.
Sarepta Therapeutics has announced that it will be working with Dr Zarife Sahenk from Nationwide Children’s Hospital in the United States to develop a gene therapy for CMT.
PTC Therapeutics recently presented positive preliminary data on the long-term clinical benefit of Translarna at the World Muscle Society conference.
Today at the World Muscle Society conference, Audentes Therapeutics announced new data from its ASPIRO study testing a gene therapy for X-linked myotubular myopathy.
Sarepta Therapeutics has released an update on the four boys dosed in its ongoing gene therapy trial.
PTC Therapeutics has released preliminary data from its ongoing clinical studies, FIREFISH and SUNFISH.
We would like to inform you that recruitment for AveXis’ STRIVE-EU gene therapy trial has started at Great Ormond Street Hospital.