New article published on “Therapies for Inherited Retinal Dystrophies: What is Enough?”
A new article of ERN-EYE has been published in Drug Discovery Today. This article, titled “Therapies for Inherited Retinal Dystrophies: What is Enough?”, describes the challenges faced when bringing new treatments to patients with inherited retinal dystrophies (IRDs), and outlines possible solutions for both the IRD and the wider rare diseases community to explore.
Context
IRDs are rare, genetically complex diseases leading to progressive vision loss and blindness, affecting over four million people globally. Following 20 years of research, the first gene therapy, Voretigene Neparvovec (Luxturna®), was approved, improving life quality for those with RPE65 mutations. However, recent challenges in clinical trials have highlighted issues such as small patient populations, complex trial designs, and the need for innovative outcome measures. To succeed, future trials must adopt tailored approaches, integrate patient perspectives, and ensure global regulatory harmonization to bring effective IRD therapies to market.
Highlights
- IRDs affect over 4M people worldwide, leading to blindness and high societal cost
- Despite a diversity of therapeutic approaches, many clinical trials have been abruptly halted
- This raises key questions, e.g. what is a meaningful impact on patients’ daily life?
- Identifying reliable outcome measures for all actors, including patients, is urgent
- Rare disease clinical trials need specifically designed studies and global regulatory pathways
Read it now!
The full article is available on the link below.