Research news

The US FDA has granted approval to Zolgensma, the first gene therapy for SMA. Although the approval process in Europe is separate to the US, it’s a major step forward.

MDUK-funded researchers have corrected a common mutation causing Ullrich congenital muscular dystrophy in the laboratory.

The first set of care recommendations for congenital and childhood-onset myotonic dystrophy type 1, and myotonic dystrophy type 2 have been published.

The drug company Dynacure has announced today that UK regulators have approved its UNITE-CNM phase I/II clinical trial.

To participate in the new study, you’ll simply need to live your life as usual and complete some questionnaires as well as wear an activity monitor for certain periods.

Fulcrum Therapeutics has obtained rights to losmapimod from GSK. Fulcrum is interested in the drug because it has found that it can switch off DUX4 in FSHD cells.

Capricor Therapeutics has recently published the final results from its HOPE-Duchenne clinical trial. This study evaluated the safety and efficacy of a cell therapy called CAP-1002

The trial results so far show that children treated with Zolgensma are living longer and achieving significant development milestones.

Wave Life Sciences has shared details of its upcoming Phase II/III clinical trial testing suvodirsen in boys with DMD. Suvodirsen is an exon-skipping drug that targets exon 51.

The 12th UK Neuromuscular Translational Research Conference was held in Newcastle on 4-5 April 2019.