Current Edition

Exploring the Limb Girdle Muscular Dystrophy Clinical Trial Landscape

With the recent advent of adeno-associated, virus-based gene therapy treatments, limb girdle muscular dystrophy (LGMD) is currently attracting the attention of the biopharmaceutical industry, especially with the goal of restoring full or partial proteins that are otherwise dysregulated. Raymond A. Huml, Siddharth Aras, Marie Trad, Tiffany Chow, Olja Tanjga and Jill Dawson at IQVIA, describe the results of a proactive, global LGMD feasibility study with insights from investigators currently providing LGMD patient care.