Adult polyglucosan body disease (APBD). research hub

A phase 1, double-blind, placebo-controlled, randomized, single ascending dose and multiple ascending dose study to assess the safety, tolerability, and pharmacokinetics of GHF-201 in healthy adult volunteers

This study is testing a new medicine called GHF-201 in healthy adult volunteers. It's the first step to see if the medicine is safe, how well people tolerate it, and how it moves through the body. This is important research for a condition called Adult Polyglucosan Body Disease.