Rare Disease and Pediatrics Expert to Address WODC Europe November 7

DURHAM, N.C., October 30, 2018 — Angi Robinson, Executive Director for Rare Disease and Pediatrics at Premier Research, will discuss clinical development strategies for genetic rare disease research November 7 at the World Orphan Drug Congress in Barcelona. Ms. Robinson’s presentation, starting at 2:20 p.m., will examine management of confirmatory testing and specialty labs, the...

Clinical Research: Phase 1 - Phase 4

Fragile X: The Quest to Treat a Complex, Little-Understood Condition

We worked on a Phase II study of a drug to treat Fragile X syndrome that, like most rare work we do, was a challenge from the start. Fragile X is a rare and not fully understood genetic disorder, typically resulting from an expansion of the CGG triplet repeat within the Fragile X mental retardation 1 (FMR1) gene...