A Phase 1b, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease.

This document provides information about a clinical trial for the treatment of Pelizaeus-Merzbacher Disease (PMD). The trial is testing a drug called ION356, which is administered through intrathecal injection. The trial has two experimental cohorts, each receiving a different dose of ION356. The study aims to enroll 24 male participants between the ages of 2 and 17 years. The estimated completion date for the trial is June 2028. The document also includes eligibility criteria and contact information for the study. [Extracted from the article]