Rare and Neglected Diseases Archives

Case Study

Simcyp’s Age-Defining Feature Facilitates Everolimus Dosing in Neonates

Everolimus (Afinitor®) was recently approved as adjunctive therapy for tuberous sclerosis complex (TSC)-associated partial seizures…

CertaraAugust 26, 2021

Case Study

Eliglustat for Gaucher Disease: Quantifying the Impact of Pharmacogenetic Status on DDIs

In 2014, Eliglustat (Cerdelga®) was approved by the FDA as the first long-term treatment for…

CertaraAugust 26, 2021

Case Study

Using PBPK for Label Recommendations in Rare Disease: Deflazacort for Duchenne Muscular Dystrophy

CertaraAugust 20, 2021

Case Study

DDI Regulatory Poster Child using the Simcyp Simulator: Ibrutinib (Imbruvica®)

Pharmacylics and J&J sought to bring ibrutinib, its new tyrosine kinase inhibitor therapy targeting rare…

CertaraJuly 29, 2021

Case Study

Voxelotor for Treating Sickle Cell Disease: Leveraging Dose Prediction Model for DDI Prediction Without Any Clinical Studies

In November 2019, the US FDA granted accelerated approval for Oxbryta™ (voxelotor) tablets for the…

CertaraJuly 14, 2021

On-Demand Webinar

The Promises and Pitfalls of Gene Therapy for Rare Diseases

The state of gene therapy faces much uncertainty as recent regulatory and clinical setbacks have…

CertaraApril 29, 2021

On-Demand Webinar

The Rare Disease Drug Dilemma: Great Value, High Price, Limited Access?

This webinar will explain how pricing has evolved for the rare disease drug market and…

CertaraFebruary 19, 2020

On-Demand Webinar

Innovative Solutions for Fast-Tracking Drugs for Rare Diseases

This webinar discussed which pharmaceutical payer strategies work and which don't in our rapidly changing…

CertaraAugust 10, 2019

Publication

A Randomized, First-in-human, Healthy Volunteer Trial of Sutimlimab, a Humanized Antibody for the Specific Inhibition of the Classical Complement Pathway

CertaraMay 8, 2018

On-Demand Webinar

Obeticholic Acid—From PK Model to Drug Label

Watch this webinar with Jeffrey Edwards to learn how he used physiologic pharmacokinetic modeling to…

CertaraJune 1, 2017

Rare and Neglected Diseases

Simcyp’s Age-Defining Feature Facilitates Everolimus Dosing in Neonates

Eliglustat for Gaucher Disease: Quantifying the Impact of Pharmacogenetic Status on DDIs

Using PBPK for Label Recommendations in Rare Disease: Deflazacort for Duchenne Muscular Dystrophy

DDI Regulatory Poster Child using the Simcyp Simulator: Ibrutinib (Imbruvica®)

Voxelotor for Treating Sickle Cell Disease: Leveraging Dose Prediction Model for DDI Prediction Without Any Clinical Studies

The Promises and Pitfalls of Gene Therapy for Rare Diseases

The Rare Disease Drug Dilemma: Great Value, High Price, Limited Access?

Innovative Solutions for Fast-Tracking Drugs for Rare Diseases

A Randomized, First-in-human, Healthy Volunteer Trial of Sutimlimab, a Humanized Antibody for the Specific Inhibition of the Classical Complement Pathway

Obeticholic Acid—From PK Model to Drug Label

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