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.
New study for Dysferlinopathy (LGMD2B/R2 or MM) is looking for participants
The second phase of an LGMD 2B study is looking for participants
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IBM drug receives Fast Track designation
19/12/2019Orphazyme have announced that the U.S Food and Drug Administration (FDA) have granted the drug, arimoclomol, Fast Track designation.
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Duchenne exon skipping drug approved by FDA
13/12/2019Sarepta’s exon skipping drug, VYONDYS 53™ (golodirsen) has been approved by the Food and Drug Administration (FDA). The FDA regulates drugs in the USA.
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UNITE-DMD update
09/12/2019Preparatory work for the clinical trial is underway and progress is being made towards the development of next-generation microdystrophins.
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Occupational Therapy Research Survey
04/11/2019RCOT 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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Positive results from LGMD gene therapy trial
04/10/2019Sarepta Therapeutics have announced new data from its trial testing SRP-9003, a gene therapy for limb girdle muscular dystrophy (LGMD) type 2E.
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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).