Novotech Publishes Duchenne Muscular Dystrophy Landscape Report to Support Biotech Research Planning 

Boston, USA - Novotech, the global full-service clinical Contract Research Organization (CRO) that partners with biotech companies to accelerate the development of advanced and novel therapeutics at every phase, has released today an industry-leading report on Duchenne Muscular Dystrophy (DMD) Clinical Trial Landscape.  

The report includes emerging genetic therapies, funding landscape, and a SWOT analysis designed to inform strategic drug development decision-making and ultimately enhancing the chances of successful and impactful treatments for DMD. The Novotech research analyst team provides these expert reports monthly, completely free of charge. These reports offer current insights into global clinical trial activity, revealing which regions experience the highest trial volumes and the factors behind these trends. They tackle the hurdles faced by biotech firms in specific therapeutic areas and discuss future paths in therapy and investment trends. 

The Duchenne Muscular Dystrophy - Global Clinical Trial Landscape report examines the characterizations of DMD: it’s the most prevalent form of muscular dystrophy, it has a higher prevalence in males, the lack of dystrophin protein (caused by a mutation) leads to deterioration of muscle health, and the age DMD presents itself which is between 3-6 years. The disorder affects 1 in 5,000 live male births, with about 20,000 new global diagnoses annually, and is usually inherited through the mother, but 25% of cases occur spontaneously without any family history. 

The report also focuses on the epidemiology, therapeutic challenges, and the evolving clinical trial trend data, including treatments and funding, offering invaluable insights for healthcare professionals and researchers.  

Management of the disease currently focuses on addressing symptoms and slowing disease progression whereas current research focuses more on therapeutic approaches including gene replacement, cell therapy, and membrane stabilization. 

The global prevalence of this complex genetic disorder varies across regions. Europe leads with the highest prevalence, followed by North America, APAC and the Rest of World (ROW). Within Europe, countries such as Sweden, Norway, and Estonia present higher than-average rates of diagnosis at 16.7 and 16.2, and 12.8 cases per 100,000, respectively. In North America, Canada and the US sit at 10.6 and 10.2. In APAC China leads at 10.3 cases per 100,000. 

Novotech’s research found there have been approximately 300 clinical trials initiated for DMD globally since 2019. North America leads in conducting trials (37%), followed by Asia Pacific (30%), Europe (24%) and ROW (9%).  

Key takeaways: 

• Analyzing patient recruitment trends since 2019 in single-country DMD clinical trials, where Europe consistently shows shorter recruitment durations and faster rates compared to the US and Asia Pacific. This may be attributed to streamlined regulatory processes, greater patient awareness, and more centralized healthcare systems. 
• There is a strong combination of public and venture-based funding for DMD out of the US in particular, but notable investments have been made in UK and South Korea as well, with the majority of funding concentrated on the preclinical stage. 
• Current research for DMD explores gene and RNA therapies with stand outs specifically for membrane stabilization techniques and interventions targeting secondary cascades as promising areas. 
• There is a robust DMD drug development pipeline with 28 drugs in preclinical stages, 14 in Phase I, 14 in Phase II, and 9 in Phase III. 

This comprehensive resource guides healthcare professionals, researchers, and organizations in navigating the complex clinical trial DMD landscape.  

Download the report here