Rare and Neglected Diseases Archives

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…

Jim GallagherApril 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…

Danielle PillsburyFebruary 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…

Danielle PillsburyAugust 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

Danielle PillsburyMay 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…

Danielle PillsburyJune 1, 2017

Publication

Modeling and Experimental Studies of Obeticholic Acid Exposure and the Impact of Cirrhosis Stage

Danielle PillsburyDecember 1, 2016

Brochure

Rare Disease and Orphan Drug Development

There are 7,000 rare diseases impacting 350 million patients worldwide, yet only 300 of these…

Danielle PillsburyAugust 3, 2016

Publication

Physiologically-based Pharmacokinetic Modeling in Pediatric Oncology Drug Development

Danielle PillsburyJuly 1, 2016

Publication

Population Pharmacokinetic and Pharmacodynamic Analyses from a 4-Month Intra-dose Escalation and Its Subsequent 12-month Dose Titration Studies for a Human Monoclonal Anti-FGF23 Antibody (KRN23) in Adults with X-linked Hypophosphatemia

X-linked hypophosphatemia (XLH) is an inherited metabolic bone disease with abnormally elevated serum FGF23 resulting…

Danielle PillsburyApril 1, 2016

Publication

Chemotherapy of Second Stage Human African Trypanosomiasis: Comparison Between the Parenteral Diamidine DB829 and Its Oral Prodrug DB868 in Vervet Monkeys

Human African trypanosomiasis (HAT, sleeping sickness) ranks among the most neglected tropical diseases based on…

Danielle PillsburyFebruary 15, 2015

Rare and Neglected Diseases

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

Modeling and Experimental Studies of Obeticholic Acid Exposure and the Impact of Cirrhosis Stage

Rare Disease and Orphan Drug Development

Physiologically-based Pharmacokinetic Modeling in Pediatric Oncology Drug Development

Population Pharmacokinetic and Pharmacodynamic Analyses from a 4-Month Intra-dose Escalation and Its Subsequent 12-month Dose Titration Studies for a Human Monoclonal Anti-FGF23 Antibody (KRN23) in Adults with X-linked Hypophosphatemia

Chemotherapy of Second Stage Human African Trypanosomiasis: Comparison Between the Parenteral Diamidine DB829 and Its Oral Prodrug DB868 in Vervet Monkeys

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