VISION-DMD project. Vamorolone - An innovative steroid like intervention for DMD
Published November 10, 2021 | Version v2
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Future clinical trial design and innovative end points (Biomarkers and Imaging) for DMD and other Neuromuscular diseases workshop report 2019

Creators

  • 1. Ceratium Ltd
  • 2. Critical Path Institute
  • 3. Collaborative Trajectory Analysis Project
  • 4. Leiden UMC

Description

The workshop was held on 29th November 2018 at Motol University Hospital Prague and was a co-run event by the VISION-DMD project and the European Reference Network for Neuromuscular Disease EURO-NMD.

The first part of the workshop summarised the state of the art with presentations from leading experts from industry, academia, regulators, patient foundations and the clinical specialists and was attended by over 50 participants. Following the presentations an expert panel discussion with invited participants addressed the current landscape and future aims and objectives. A follow-up discussion (January 2019, Leiden), future planning and summary of biomarker data collated so far are also described.

Files

Future clinical trial design and innovative end points (Biomarkers and Imaging) for DMD and other Neuromuscular diseases workshop report.pdf

Files (1.0 MB)

Additional details

Funding

VISION DMD – VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy 667078
European Commission