The FDA’s emerging framework represents more than a regulatory update; it is a paradigm shift toward human-relevant, ...

Well before a human receives the first dose of an experimental drug, it’s evaluated in an animal to test for toxicity. Monkeys are often chosen for their genetic similarities to humans, and safe ...

An Ionis Pharmaceuticals drug in development for Angelman syndrome has mid-stage clinical trial results showing improvement across a range of measures of this rare neurodevelopmental disorder, and the ...

Imbria Pharmaceuticals’ heart failure medicine met the safety and tolerability goals in a phase 2 trial while also improving results on a cardiopulmonary exercise test, with patients reporting other ...

The FDA has cleared the initiation of a pivotal Phase 3 trial testing pridopidine in people with early, rapidly progressive ...