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.
Highlights of the 2021 Neuromuscular Translational Research Conference
The conference brought together scientists and clinicians from around the world to share the latest advances in cutting-edge research.
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SMA drug Evrysdi (risdiplam) approved in the EU
30/03/2021The European Commission (EC) has granted marketing authorisation to Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA).
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Updates from the FSHD, myotonic dystrophy, Col6- dystrophies and GNE myopathy registries
26/03/2021The patient registries for FSHD, DM, COL6-dystrophies and GNE myopathy have shared their latest newsletters.
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Pharnext shares update on its clinical development of CMT1A drug
24/03/2021The pharmaceutical company Pharnext is on track to start its phase III clinical trial, PREMIER in the USA, by the end of March 2021.
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Muscular Dystrophy UK launches 2021 research grant round
09/03/2021Muscular Dystrophy UK is delighted to announce the launch of its 2021 grant round.
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SMA gene treatment Zolgensma accepted for restricted use within NHS Scotland
08/03/2021SMA gene treatment Zolgensma accepted for restricted use within NHS Scotland
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Four charities call on SMC to approve treatment for Duchenne muscular dystrophy
02/03/2021A group of charities, including Muscular Dystrophy UK, is urging for the approval of a drug that can treat an underlying genetic cause of a life-limiting muscle-wasting condition.
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CHMP gives positive opinion on Evrysdi (risdiplam) for the treatment of SMA
26/02/2021EMA committee gives positive opinion on Evrysdi (risdiplam) for the treatment of SMA
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First gene therapy trial for DMD to start recruitment in the UK
25/02/2021For the first time, a clinical trial will start recruiting a small number of boys in the UK to test a potential gene therapy for the treatment of DMD.
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International Mitochondrial Disease Covid-19 Data Study
05/02/2021The research team hopes that this study will help to understand how Covid-19 affects people with mitochondrial disorders
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Survey reveals how Covid-19 has affected activity levels of disabled people
03/02/2021The findings come following a survey from Activity Alliance, a charity working to make active lives possible for disabled people.