Dmd/bmd – outcome measures

Funded in 2015, the DMD Hub, a collaboration between Duchenne UK and the two UK neuromuscular centres of excellence, is an established network of Duchenne muscular dystrophy (DMD) clinical trial sites in the UK (www.dmdhub.org). With the arrival of advanced therapy trials for DMD, the DMD Hub is supporting the neuromuscular community to facilitate the setting up and running these trials in the UK. In November 2019, the DMD Hub held the first gene therapy meeting with over 100 key stakeholders including clinical experts, patient representatives, industry, regulators and payers to discuss existing barriers to gene therapy access through clinical trials and as approved treatment in the UK.

Source: Neuromuscular Disorders - September 19, 2021 Category: Neurology Authors: E. Heslop, C. Turner, E. George, A. Irvin, A. Robertson, E. Crossley, A. Johnson, R. Fischer, H. Peay, F. Muntoni, V. Straub, M. Guglieri Source Type: research