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.
MDUK-funded research has shown that a new type of molecular patch could be promising for DM1.
Santhera Pharmaceuticals has announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has designated vamorolone as Promising Innovative Medicine (PIM) f
Facio Therapies has announced the completion of a ‘real-world data’ study monitoring the progression of FSHD and its impact on daily life.
Sarepta Therapeutics have announced new data from its trial testing SRP-9003, a gene therapy for limb girdle muscular dystrophy (LGMD) type 2E.
PTC Therapeutics today announced data demonstrating the ongoing benefit of risdiplam for the treatment of all types of spinal muscular atrophy (SMA).
Pharmaceutical company, Catabasis, and American charity Jain Foundation have announced a preclinical research collaboration to study edasalonexent in dysferlinopathy (LGMD 2B).
Physical Activities in Rare Neurological Conditions (PARC) is a study aiming to support physical activity for people with rare neurological diseases.
Acceleron Pharma has announced topline results from its phase 2 trial testing ACE-083 in adults with facioscapulohumeral muscular dystrophy (FSHD).
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.