Advancing Rare Disease Therapies: The Power of Regulatory Expertise
Bringing treatments to patients with rare diseases has long been one of the biggest challenges…
Certara2025 年 2 月 26 日
How Biosimulation is Transforming Rare Disease Drug Development
Developing therapeutics for rare diseases comes with unique challenges—small patient populations, high variability in treatment…
Certara2025 年 2 月 26 日
Gene Therapy: A Transformative Shift in Rare Disease Treatment
After decades of promise, gene therapy is finally starting to deliver on its potential—especially for…
Certara2025 年 2 月 26 日
Navigating Rare Disease Regulations: Challenges, Opportunities, and Innovative Solutions
Bringing life-changing therapies to rare disease patients requires expertise, innovation, and a deep understanding of…
Certara2025 年 2 月 26 日
Why FDA’s omaveloxolone approval for Friedreich’s ataxia matters
2025 年 1 月 10 日 Friedreich’s Ataxia (FA) is a rare, genetic, and progressive neurodegenerative disorder. A…
Certara2025 年 1 月 10 日
Quantitative Systems Pharmacology-Based Digital Twins Approach Supplements Clinical Trial Data for Enzyme Replacement Therapies in Pompe Disease
Pompe disease is a rare and complex condition with severe infantile-onset (IOPD) and late-onset (LOPD)…
Certara2024 年 12 月 4 日
New Horizons of Model Informed Drug Development in Rare Diseases Drug Development
Model-informed drug development is revolutionizing the way we approach rare diseases, where traditional large-scale clinical…
Certara2024 年 7 月 11 日
Advancing Therapeutic Development in Rare Diseases: Overcoming Key Challenges with Model-Informed Strategies
Read this white paper by Certara's expert Dr. Rajesh Krishna and learn about the core…
Certara2024 年 4 月 18 日
