Rare and Neglected Diseases Archives

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…

CertaraFebruary 26, 2025

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…

CertaraFebruary 26, 2025

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…

CertaraFebruary 26, 2025

Fact Sheet

The Tools You Need to Accelerate Rare Disease Drug Development

CertaraFebruary 20, 2025

Blog

Why FDA’s omaveloxolone approval for Friedreich’s ataxia matters

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

CertaraJanuary 10, 2025

Poster

Population Pharmacokinetics (PopPK) of Revumenib in Patients with Relapsed/Refractory Acute Leukemias

CertaraDecember 12, 2024

Publication

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)…

CertaraDecember 4, 2024

Publication

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…

CertaraJuly 11, 2024

Blog

How to Navigate the FDA Landscape for an Orphan Drug

Rare diseases are a public health priority. FDA has launched several programs and a final…

CertaraFebruary 21, 2024

Blog

Methods to Increase Signal Finding in Rare Disease Drug Development

The regulatory landscape for drugs to treat rare diseases is being disrupted. Gleaning from several…

CertaraSeptember 8, 2023

Rare and Neglected Diseases

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

The Tools You Need to Accelerate Rare Disease Drug Development

Why FDA’s omaveloxolone approval for Friedreich’s ataxia matters

Population Pharmacokinetics (PopPK) of Revumenib in Patients with Relapsed/Refractory Acute Leukemias

Quantitative Systems Pharmacology-Based Digital Twins Approach Supplements Clinical Trial Data for Enzyme Replacement Therapies in Pompe Disease

New Horizons of Model Informed Drug Development in Rare Diseases Drug Development

How to Navigate the FDA Landscape for an Orphan Drug

Methods to Increase Signal Finding in Rare Disease Drug Development

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