Rare and Neglected Diseases Archives

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A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease

New therapeutics discovery and development for ocular diseases have been traditionally associated with a low…

CertaraDecember 15, 2021

Case Study

Dose Optimization Using Population PK for an Orphan Drug

RAPALIMUS® Tablets (sirolimus) is Nobelpharma’s orphan drug developed from an oral medication that is sold…

CertaraNovember 17, 2021

On-Demand Webinar

The Scary Future of Rare Disease Management

In a world of rising healthcare costs, the greatest nightmare for payers is reimbursing drug…

CertaraSeptember 23, 2021

Case Study

Using a PBPK-PD Receptor Occupancy Model to Determine the Optimal Dose of Radiprodil for Neonates

Radiprodil is a selective allosteric modulator of the NR2B N-methyl-D-aspartate receptor GluN2B-NMDA that was being…

CertaraAugust 26, 2021

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

Rare and Neglected Diseases

A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease

Dose Optimization Using Population PK for an Orphan Drug

The Scary Future of Rare Disease Management

Using a PBPK-PD Receptor Occupancy Model to Determine the Optimal Dose of Radiprodil for Neonates

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

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