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
Work has begun in Professor George Dickson’s lab to refine the gene therapy product.
Wave Life Sciences has announced positive results from its phase 1 clinical trial testing WVE-210201.
Pharnext is carrying out an observational study for people with Charcot-Marie Tooth disease using a new digital app called CMT&Me.
Novartis has announced that the US Food and Drug Administration (FDA) has accepted its marketing application for AVXS-101 for the treatment of SMA type 1.
The US Food and Drug Administration has granted Fast Track Designation to ACE-083 for the treatment of individuals with Charcot-Marie-Tooth Disease (CMT).
A recent study has demonstrated the value of a new mouse model of facioscapulohumeral muscular dystrophy (FSHD).
Stealth BioTherapeutics has initiated a phase 3 trial to test elamipretide in individuals with primary mitochondrial myopathy.
Solid is continuing to enrol boys into IGNITE DMD and expects to release preliminary trial results in the first quarter of 2019.
Myonexus Therapeutics has initiated the first-ever gene therapy (MYO-101) trial for people with limb girdle muscular dystrophy type 2E (LGMD 2E).
An international team of experts have published consensus-based care recommendations to help clinicians deliver high standards of care to adults with myotonic dystrophy 1.