Rare and Neglected Diseases Archives

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Certara’s Virtual Twin® QSP Models: Transforming Rare Disease Research into Clinical Insights

Certara’s Virtual Twin® QSP Models are transforming rare disease drug development, with real-world insights from…

Certara2025 年 11 月 25 日

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…

Certara2025 年 11 月 12 日

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…

Certara2025 年 11 月 10 日

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 Pillsbury2025 年 9 月 10 日

Case Study

杜氏肌营养不良症（DMD）药物研发中的剂量优化与监管结果优化

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

Certara2025 年 7 月 29 日

Press Coverage

数字孪生与虚拟试验：重塑罕见病药物研发新范式

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

Certara2025 年 7 月 22 日

White Paper

罕见肿瘤学产品发布手册：在美国和欧洲取得成功的通用策略

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Certara2025 年 2 月 28 日

Video

加快罕见病药物开发中的 CMC 成功率

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

Certara2025 年 2 月 26 日

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.…

Certara2025 年 2 月 26 日

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…

Certara2025 年 2 月 26 日

罕见病和被忽视疾病

Certara’s Virtual Twin® QSP Models: Transforming Rare Disease Research into Clinical Insights

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

杜氏肌营养不良症（DMD）药物研发中的剂量优化与监管结果优化

数字孪生与虚拟试验：重塑罕见病药物研发新范式

罕见肿瘤学产品发布手册：在美国和欧洲取得成功的通用策略

加快罕见病药物开发中的 CMC 成功率

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

Bridging Global Health Disparities in Rare Disease Drug Development

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