UK Rare Diseases Framework published
The UK Government and the devolved administrations have published a new UK Rare Diseases Framework.
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AMO Pharma announces clinical trial for AMO-02 in the treatment of Congenital Myotonic Dystrophy
12/01/2021AMO-Pharma announces the initiation of Phase 2/3 clinical trial, REACH-CDM.
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Impact of Brexit trade deal on medicines, healthcare rights and collaboration
08/01/2021Brexit will have a wide ranging impact on the lives of everyone, including people living with a muscle-wasting condition.
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NICE statement on pause to re-evaluation of Translarna
23/12/2020NICE statement on pause to re-evaluation of Translarna
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Cambridge University Hospitals NHS Trust recommends use of risdiplam
23/12/2020Cambridge University Hospitals NHS Trust recommends use of risdiplam
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Consultation response to NICE Methods Review
21/12/2020MDUK submitted a consultation response to a Methods Review by NICE, which focuses on the way it evaluates new treatments.
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Important announcement for adults with Duchenne muscular dystrophy
18/12/2020Muscular Dystrophy UK is delighted to announce the successful completion of two projects we funded to improve care and support for adults living with Duchenne muscular dystrophy.
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Update on stoppage of SIDEROS clinical trial for Raxone/idebenone
18/12/2020Update on stoppage of SIDEROS clinical trial for Raxone/idebenone
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PepGen secures $45m investment to drive molecular patches to clinic
10/12/2020PepGen’s work into molecular patches seeks to help people with Duchenne muscular dystrophy (DMD) and other muscle-wasting conditions.
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Green light for a Duchenne muscular dystrophy gene therapy in France
07/12/2020These are exciting times for the development of treatments, some of which have the potential to markedly improve outcomes for a number of neuromuscular conditions.
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SMC approves mexiletine hydrochloride for adults with non-dystrophic myotonic disorders
07/12/2020The news means adult patients in Scotland will be able to access mexiletine hydrochloride on the NHS if their healthcare professional thinks it is right for them.