Research news

A landmark trial has shown that cognitive behavioural therapy (CBT) can benefit people with myotonic dystrophy type 1.

Acceleron has announced the FDA has granted Orphan Drug Designation to ACE-083 for the treatment of FSHD.

We are disappointed to hear that Summit Therapeutics has decided to discontinue the development of its utrophin modulator, ezutromid.

Myonexus Therapeutics gave an update on its LGMD gene therapy programmes at Sarepta’s recent R&D day.

Sarepta Therapeutics announced positive preliminary results from its ongoing Duchenne gene therapy trial at the company’s R&D day.

A £35,000 investment into myotonic dystrophy at St Andrews University will ‘bring hope’ to those living with it, says Diana, Countess of Lindsay.

A new study has explored parents' perspectives on clinical trials and what influences their decision to enrol their child.

The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended that the current licence for Translarna be expanded to include boys aged 2-5 years.

Audentes Therapeutics has provided an update on its ASPIRO gene therapy trial. The study is evaluating AT132 in young children with X-linked myotubular myopathy.

Researchers at University College London (UCL) have developed artificial ‘mini-muscles’ that could speed up testing potential treatments for muscle-wasting conditions.