Highlights The issue of translatability of pre-clinical results was discussed with a wide range of stakeholders. Challenges and pitfalls …
Action Duchenne CEO represents the community at ENMC International WorkshopRead More
NICE guideline publication – Adult social care: improving people’s experience
NICE guideline on people's experience in adult social care services: improving the experience of care and support for people using adult social care …
NICE guideline publication – Adult social care: improving people’s experienceRead More
Summit announces new analysis showing ezutromid significantly reduced muscle inflammation
Summit has announced further positive findings from PhaseOut DMD, a Phase 2 open-label, multi-centre clinical trial of the utrophin modulator …
Summit announces new analysis showing ezutromid significantly reduced muscle inflammationRead More
Skeletal and cardiac muscle improvements in MDX mouse
Cedars-Sinai Researchers Say Findings Lay Groundwork for Testing of CAP-1002 in Patients with Duchenne Muscular Dystrophy Capricor have today …
Skeletal and cardiac muscle improvements in MDX mouseRead More
Wales joins the 100,000 Genomes Project
Wales has joined the 100,000 Genomes Project, the Welsh Government announced. The project, now a UK-wide initiative, seeks to transform patient …
PTC receives formal dispute resolution request decision from the FDA
PTC have today announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA's prior …
PTC receives formal dispute resolution request decision from the FDARead More
FDA share guidance for Duchenne and Becker muscular dystrophy drug development
We are happy to share the news that the U.S. Food and Drug Administration (FDA) have published a guidance document for industry, which details what …
FDA share guidance for Duchenne and Becker muscular dystrophy drug developmentRead More
Summit discuss their recently reported positive data from their ‘PhaseOut DMD’ trial (webinar)
Download the FAQ Bringing you the most up to date news from the international Duchenne community is one of our core aims. Action Duchenne and …
Catabasis reports Edsalonexent preserved muscle function and slowed progression of Duchenne through more than one year of treatment
Catabasis have reported new positive efficacy and safety results showing preservation of muscle function and sustained condition-modifying effects in …
Capricor receives FDA RMAT designation for CAP-1002
Capricor have announced that the U.S. Food and Drug Administration (FDA) has granted CAP-1002 the Regenerative Medicine Advanced Therapy (RMAT) …
Capricor receives FDA RMAT designation for CAP-1002Read More
Investigational idebenone becomes available through EAP in US
Santhera announces the launch of a U.S. Expanded Access Program (EAP) referred to as BreatheDMD with idebenone for patients with DMD. Through the …
Investigational idebenone becomes available through EAP in USRead More
TREAT-NMD steroids analysis demonstrates the power of ‘real-world’ Registry data
This study underlines the importance of data collection via patient registries and the critical role of multicenter collaboration in the rare disease …
TREAT-NMD steroids analysis demonstrates the power of ‘real-world’ Registry dataRead More
