News, blogs and stories

Read the latest news, browse other people's stories and find blogs about experiences you relate to. You can also read our publications like Target MD, and find our most recent Press Releases.

Advanced filters

News
We were joined by Prof Mary Reilly (Professor of clinical Neurology at UCL) who spoke about the…
News
To mark Becker awareness week, we were joined in our BMD seminar by researcher Federica Montanaro,…
News
There are currently no approved drugs for Charcot-Marie-Tooth disease (CMT), but clinical trials…
News
Orphan drug status for treatment of myotonic dystrophy type 1   With no cures for myotonic…
News
SMA UK, MDUK and TreatSMA have today written to the Scottish Medicines Consortium (SMC) to raise…
News
This study is an extension of a phase 2 clinical trial in which 16 boys with Duchenne muscular…
News
The results of the trial showed that people taking the drug have a slowing of disease progression…
News
Risdiplam is an oral drug that increases SMN protein levels by targeting the SMN2 gene manufactured…
News
Muscular Dystrophy UK is funding this research, looking into how dystrophin works in the heart and…