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.
Occupational Therapy Research Survey
RCOT has launched a survey to identify the top 10 research priorities for occupational therapy in the UK. More information can be found at the RCOT website: www.rcot.co.uk/otpsp
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University College London researcher wins MDUK’s first Research Image Competition
01/11/2019Senior Research Fellow Dr James Sleigh as the winner of our first Research Image Competition.
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Molecular patches show promise in a mouse model of myotonic dystrophy type 1
25/10/2019MDUK-funded research has shown that a new type of molecular patch could be promising for DM1.
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Real-world data study in FSHD
15/10/2019Facio Therapies has announced the completion of a ‘real-world data’ study monitoring the progression of FSHD and its impact on daily life.
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Update on oral SMA drug
02/10/2019PTC Therapeutics today announced data demonstrating the ongoing benefit of risdiplam for the treatment of all types of spinal muscular atrophy (SMA).
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Exploring physical activity – opportunity to take part in research
24/09/2019Physical Activities in Rare Neurological Conditions (PARC) is a study aiming to support physical activity for people with rare neurological diseases.
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Myasthenia gravis drug receives Orphan Drug Designation
05/09/2019Ra Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to zilucoplan for the treatment of patients with generalised mya
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£1.2m invested in 9 new research projects for 2019
02/09/2019Muscular Dystrophy UK is investing £1.2 million into 9 new research projects this year, bringing our total research portfolio to over £10m.
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Genetic factors that determine the severity of myotonic dystrophy type 1
23/08/2019Researchers involved in the OPTIMISTIC trial have published a scientific paper that identifies genetic factors that influence the severity of myotonic dystrophy type 1.
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FDA rejects Sarepta’s exon 53 drug
20/08/2019The US Food and Drug Administration (FDA) has issued a Complete Response Letter to Sarepta Therapeutics in relation to its application for approval of Duchenne drug golodirsen.
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Audentes update on neuromuscular programmes
07/08/2019Audentes Therapeutics has provided an update on its drugs in development for myotubular myopathy, Duchenne muscular dystrophy, myotonic dystrophy type 1 and Pompe disease.