A centre of expertise in muscle
- 26 July 2019 European regulators’ views on a wearable-derived performance measurement of ambulation for DMD regulatory trials
- 26 July 2019 Long-term natural history data in DMD ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53
- 25 July 2019 Development and validation of a new risk prediction score for life-threatening ventricular tachyarrhythmias in Laminopathies
- 25 July 2019 66 participants: all time record for our 2019 Summer School!
- 22 July 2019 Neurochemical markers in CSF of adolescent and adult SMA patients undergoing nusinersen treatment
- 10 July 2019 Impact of age and motor function in a phase 1/2A study of infants with SMA type 1 receiving single-dose gene replacement therapy