Rare and Neglected Diseases Archives

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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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Danielle Pillsbury2025 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.…

Danielle Pillsbury2025 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…

Danielle Pillsbury2025 年 2 月 26 日

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…

Danielle Pillsbury2025 年 2 月 26 日

Video

How Biosimulation is Transforming Rare Disease Drug Development

Developing therapeutics for rare diseases comes with unique challenges—small patient populations, high variability in treatment…

Danielle Pillsbury2025 年 2 月 26 日

Video

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…

Danielle Pillsbury2025 年 2 月 26 日

Video

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…

Danielle Pillsbury2025 年 2 月 26 日

Fact Sheet

加速罕见病药物开发所需的工具

Danielle Pillsbury2025 年 2 月 20 日

Blog

为何 FDA 批准 omaveloxolone 用于治疗弗里德赖希共济失调症意义重大

2025 年 1 月 10 日 Friedreich’s Ataxia (FA) is a rare, genetic, and progressive neurodegenerative disorder. A…

Danielle Pillsbury2025 年 1 月 10 日

罕见病和孤儿病

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

Advancing Rare Disease Therapies: The Power of Regulatory Expertise

How Biosimulation is Transforming Rare Disease Drug Development

Gene Therapy: A Transformative Shift in Rare Disease Treatment

Navigating Rare Disease Regulations: Challenges, Opportunities, and Innovative Solutions

加速罕见病药物开发所需的工具

为何 FDA 批准 omaveloxolone 用于治疗弗里德赖希共济失调症意义重大

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