Update on Spinraza and adult services
NHS England and Patient Advocacy Groups have recently been focused on identification of hospitals with the right expertise to treat eligible adults who have SMA with nusinersen.
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The Great North Run 2019 is here. Good luck #TeamOrange!
02/09/2019All here at Muscular Dystrophy UK would like to wish the best of luck to all 175#TeamOrange runners who are taking the Great North Run this Sunday.
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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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MDUK Premiership & Championship 2019/20 previews – get to know the teams
01/09/2019Find out where the MDUK Premiership and Championships stand going into the 2019/20 season
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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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Sam swaps his suit for a running vest for the Great North Run
23/08/2019Sam, who has been a carer for his best friend, Ryan, for eight years will be taking on the Great North Run in September.
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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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Tributes paid to determined twins
07/08/2019Loving tributes have been paid to the determination of twin brothers in North East Derbyshire who lived with Becker MD and who have died within 12 months of one another.
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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.
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Muscular Dystrophy UK calls for Changing Places toilets at all Premier League clubs
07/08/2019Muscular Dystrophy UK is urging all Premier League clubs to install Changing Places toilets as the start of the 2019/20 season approaches.
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Genome editing reverses paralysis in mouse model of congenital muscular dystrophy
24/07/2019A new study has shown that editing a ‘modifier’ gene can prevent and reverse symptoms in a mouse model of congenital muscular dystrophy type 1A.