Rare and Neglected Diseases Archives

Publication

Exposure–Response Analysis of Donidalorsen for the Treatment of Hereditary Angioedema

Certara and Ionis analyzed Phase 3 OASIS-HAE data showing flexible, effective donidalorsen dosing can reduce…

CertaraNovember 12, 2025

Case Study

Supporting Ionis’ Donidalorsen BLA with Biomarker HAE Attack Rate Modeling

Discover how Certara applied exposure-response analysis and advanced modeling to optimize Donidalorsen dosing for hereditary…

CertaraNovember 10, 2025

Press Coverage

Move Away From Animal-Only Testing May Improve, Speed Rare Disease Research

The article, "Move Away From Animal-Only Testing May Improve, Speed Rare Disease Research," explores a…

Danielle PillsburySeptember 10, 2025

Case Study

Optimizing Dosing and Regulatory Outcomes in Duchenne Muscular Dystrophy (DMD) Drug Development

Certara supported DMD drug development for Givinostat, helping Italfarmaco with pharmacometric and regulatory strategy.

CertaraJuly 29, 2025

Press Coverage

Digital twins and virtual trials: A new era in rare disease drug development

Discover how using digital twins in rare disease drug development is improving precision and accelerating…

CertaraJuly 22, 2025

White Paper

Rare Oncology Product Launch Playbook: Universal Strategies for Success in the U.S. and Europe

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CertaraFebruary 28, 2025

Video

Accelerating CMC Success in Rare Disease Drug Development

New drug approvals in rare diseases are rising, bringing hope to patients. But with accelerated…

CertaraFebruary 26, 2025

Video

Leveraging Real-World Evidence (RWE) in Rare Disease Drug Development

The path to regulatory approval and market access for rare disease therapies presents unique challenges.…

CertaraFebruary 26, 2025

Video

Bridging Global Health Disparities in Rare Disease Drug Development

Rare diseases know no borders—but access to life-changing treatments remains unequal across the world. In…

CertaraFebruary 26, 2025

Video

Advancing Rare Disease Therapies: The Power of Regulatory Expertise

Bringing treatments to patients with rare diseases has long been one of the biggest challenges…

CertaraFebruary 26, 2025

Rare and Neglected Diseases

Exposure–Response Analysis of Donidalorsen for the Treatment of Hereditary Angioedema

Supporting Ionis’ Donidalorsen BLA with Biomarker HAE Attack Rate Modeling

Move Away From Animal-Only Testing May Improve, Speed Rare Disease Research

Optimizing Dosing and Regulatory Outcomes in Duchenne Muscular Dystrophy (DMD) Drug Development

Digital twins and virtual trials: A new era in rare disease drug development

Rare Oncology Product Launch Playbook: Universal Strategies for Success in the U.S. and Europe

Accelerating CMC Success in Rare Disease Drug Development

Leveraging Real-World Evidence (RWE) in Rare Disease Drug Development

Bridging Global Health Disparities in Rare Disease Drug Development

Advancing Rare Disease Therapies: The Power of Regulatory Expertise

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