News

A new study has found that boys with Duchenne muscular dystrophy on certain steroid regimes have a higher fracture risk than others.

Sarepta Therapeutics has provided an update on the four boys participating in its gene therapy trial for Duchenne muscular dystrophy.

Polaris DMD trial has started enrolling patients at Bristol Royal Hospital for Children. Other sites include London and Manchester.

Acclaimed clinical researcher, Professor Laurent Servais, has been appointed Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre.

Access to spinal muscular atrophy (SMA) treatment Spinraza featured prominently in a House of Commons debate yesterday on NICE appraisal processes for rare disease treatments.

The aim of the Mitochondrial Disease Priority Setting Partnership (PSP) is to find the most important unanswered questions about mitochondrial diseases.

Facio Therapies announced today that it has completed a comprehensive search for new FSHD drug candidates.

Trailblazers Intern Conrad Tokarczyky blogs about campaigning and the history of Trailblazers.

Researchers identify a way to improve the efficiency of genome editing technology and boost dystrophin production in a mouse model of Duchenne muscular dystrophy.

Members of the Clay Cross Probus Club come together for a charity race-night to raise funds for Muscular Dystrophy UK