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.
Researchers at Great Ormond Street Hospital (GOSH) are carrying out a research study to find out more about the emotion system of the brain in boys with Duchenne.Read more
A phase 2 clinical trial has shown that zilucoplan can reduce the severity of generalised myasthenia gravis (MG).
Catabasis has released an update on its PolarisDMD trial. This is a global phase 3 trial testing edasalonexent (CAT-1004) in boys with Duchenne muscular dystrophy.
Sarepta Therapeutics has filed an application with the US Food and Drug Administration (FDA) seeking approval of golodirsen.
Work has begun in Professor George Dickson’s lab to refine the gene therapy product.
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.
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.
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.