News & Events

SKIP-NMD Final meeting

The final SKIP-NMD meeting was held on the 28th-29th of April in London, organised by the Coordinator UCL.

42 months of the project have passed and the results are very promising. This is the last meeting before the official EU final report is submitted. Fruitful technical discussion as well as key decisions for the next steps took part in the meeting. The study enrolment has closed in June 2016, and the 96-week extension to Part 2 is on-going.

SKIP-NMD clinical trial now progressing to part 2

The first part of the SKIP-NMD clinical trial, a dose escalation phase,  was successfully completed with no significant problem for the children recruited so far, and the team obtained the go-ahead from the data safety monitoring board to initiate the second, maintenance  phase of the study. All 12 children recruited in Part 1 are now being enrolled in Part 2 and the children who until now were receiving the placebo will now start to receive the morpholino drug.

The study is furthermore ready to recruit a second group of children who will all receive the morpholino antisense at the optimal tolerated dose, for a period of at least 48 weeks.

The next important milestone of the study is now to increase the number of participants receiving the morpholino antisense drug, and a parallel natural history part of the study is also opened to children not eligible to receive this antisense oligonucleotide. Detailed muscle imaging and novel non-invasive measures of muscle strength and endurance will be studied also in these children, so that as much information as possible on these novel measures which could help this and future clinical trials, can be gathered.

Members of the SKIP-NMD consortium met in Cagliari on 25th and 26th of June 2015

The 6ht meeting of the SKIP NMD Consortium was held in Cagliari on the 25th and 26th of June 2015. This meeting marked several key milestones in the EU funded clinical trial, following the successful end of the first part of the clinical trial and was the opportunity to discuss the muscle MRI findings and their correlation to the clinical aspects of DMD, as well as the novel non-invasive outcome measures which are being trialled for the first time in this study.