These are exciting times, with fresh news about research developments appearing every week.
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The European Commission (EC) has granted marketing authorisation to Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA).
The patient registries for FSHD, DM, COL6-dystrophies and GNE myopathy have shared their latest newsletters.
The pharmaceutical company Pharnext is on track to start its phase III clinical trial, PREMIER in the USA, by the end of March 2021.
Muscular Dystrophy UK is delighted to announce the launch of its 2021 grant round.
SMA gene treatment Zolgensma accepted for restricted use within NHS Scotland
A 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.
EMA committee gives positive opinion on Evrysdi (risdiplam) for the treatment of SMA
For 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.
The research team hopes that this study will help to understand how Covid-19 affects people with mitochondrial disorders
The findings come following a survey from Activity Alliance, a charity working to make active lives possible for disabled people.