We continue to evolve into new areas where there is great unmet need and opportunity to help people living with rare diseases and their families fully live their best lives.
While the rare disease community is large, each individual rare disease impacts a small number of people. As a result, many rare diseases are not adequately studied and have few to no treatment options.
Given small patient populations and need for real-world evidence for rare diseases, direct engagement with the patient community is essential for innovation. Our ability to advance science and make a meaningful impact for patients is tied to our success in listening to and learning from patients and proactively engaging disease communities throughout the entire research and development (R&D) process.
Given the need for evidence-based trials, direct engagement with patients is essential for innovation.
Our ability to advance science through research and development, (R&D), and make a meaningful impact for patients, is tied to our ability to listen and learn from patients and proactively engage rare disease communities throughout the entire R&D process.
We continue to lead the way in rare disease clinical R&D through our patient-centric approach. Throughout the product life cycle, we work closely with patients, caregivers and the community, beginning in the earliest stages of clinical trials to generate insights that inform our discovery and development efforts.
Using patient-focused metrics to hold ourselves accountable, we ensure our clinical programs are scientifically rigorous, accessible, and address what matters most to patients and caregivers.
We conduct rigorous clinical trials and obtain marketing approvals by regulatory authorities around the world to bring safe and effective medicines to as many patients as possible.
Our work is marked by a commitment to the highest standards of preclinical and clinical research, an acute sense of urgency, and access to our investigational medicines through participation in a clinical trial.
Learn more about our current clinical trials.
Veeva ID: GL/ALL/0062