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.
Senior Research Fellow Dr James Sleigh as the winner of our first Research Image Competition.
MDUK-funded research has shown that a new type of molecular patch could be promising for DM1.
Facio Therapies has announced the completion of a ‘real-world data’ study monitoring the progression of FSHD and its impact on daily life.
PTC Therapeutics today announced data demonstrating the ongoing benefit of risdiplam for the treatment of all types of spinal muscular atrophy (SMA).
Physical Activities in Rare Neurological Conditions (PARC) is a study aiming to support physical activity for people with rare neurological diseases.
Ra Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to zilucoplan for the treatment of patients with generalised mya
Muscular Dystrophy UK is investing £1.2 million into 9 new research projects this year, bringing our total research portfolio to over £10m.
Researchers involved in the OPTIMISTIC trial have published a scientific paper that identifies genetic factors that influence the severity of myotonic dystrophy type 1.
The 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.
Audentes Therapeutics has provided an update on its drugs in development for myotubular myopathy, Duchenne muscular dystrophy, myotonic dystrophy type 1 and Pompe disease.