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.
PREFER is looking for volunteers living with myotonic dystrophy or mitochondrial disease
As part of the IMI PREFER project, we’re working with Newcastle University to conduct focus groups for people with myotonic dystrophy type 1 or mitochondrial disease.
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New study investigating how Duchenne affects the brain
29/01/2019Researchers 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.
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Positive results from myasthenia gravis trial
21/01/2019A phase 2 clinical trial has shown that zilucoplan can reduce the severity of generalised myasthenia gravis (MG).
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Update on Catabasis’ Duchenne trial
15/01/2019Catabasis 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.
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Sarepta applies for approval of second Duchenne drug
03/01/2019Sarepta Therapeutics has filed an application with the US Food and Drug Administration (FDA) seeking approval of golodirsen.
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First year update from the UNITE-DMD gene therapy trial
14/12/2018Work has begun in Professor George Dickson’s lab to refine the gene therapy product.
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New CMT app launched
06/12/2018Pharnext is carrying out an observational study for people with Charcot-Marie Tooth disease using a new digital app called CMT&Me.
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FDA fast tracks SMA gene therapy
04/12/2018Novartis 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.
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New FSHD mouse model treated with gene therapy
26/11/2018A recent study has demonstrated the value of a new mouse model of facioscapulohumeral muscular dystrophy (FSHD).
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Mitochondrial myopathy trial recruiting in the UK
15/11/2018Stealth BioTherapeutics has initiated a phase 3 trial to test elamipretide in individuals with primary mitochondrial myopathy.
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LGMD 2E gene therapy trial starts in the USA
08/11/2018Myonexus Therapeutics has initiated the first-ever gene therapy (MYO-101) trial for people with limb girdle muscular dystrophy type 2E (LGMD 2E).