Blood-based biomarker

Blood-based biomarker Developing a more informative measure of disease progression and response to new treatments Although Duchenne has been studied for many years, classic measures of disease progression rely on basic observations of movement, such as the six-minute...

Non-invasive imaging biomarker

Non-invasive imaging biomarker Reducing biopsies and improving dystrophin analysis Biopsies have become a difficult necessity for Duchenne patients who are involved in clinical trials. In addition to the stress on patients, each muscle can present disease differently,...

LTBP4

LTBP4 Targeting muscle inflammation and fibrosis With the funds raised in 2016, the DRF is tackling inflammation and fibrosis, which are two key problems for Duchenne boys. It is believed that the protein LTBP4 prevents the activation of a key biological pathway...

Vamorolone

Vamorolone Alternative to existing steroids for Duchenne patients The only current standard of care for boys with Duchenne is early treatment with long-term steroids, which gives some protection to muscles but with significant side effects. In July 2014, alongside...