Rare and Neglected Diseases Archives

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

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…

Certara2025 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…

Certara2025 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…

Certara2025 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…

Certara2025 年 2 月 26 日

Fact Sheet Infographic

加速罕见病药物开发所需的工具

Certara2025 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…

Certara2025 年 1 月 10 日

Poster

Revumenib 在复发性/难治性急性白血病患者中的群体药代动力学 (PopPK)

Certara2024 年 12 月 12 日

Publication

基于定量系统药理学的数字双胞胎方法补充了庞贝氏症酶替代疗法的临床试验数据

Pompe disease is a rare and complex condition with severe infantile-onset (IOPD) and late-onset (LOPD)…

Certara2024 年 12 月 4 日

罕见病和被忽视疾病

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

Revumenib 在复发性/难治性急性白血病患者中的群体药代动力学 (PopPK)

基于定量系统药理学的数字双胞胎方法补充了庞贝氏症酶替代疗法的临床试验数据

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