Aceragen is a rare disease biopharmaceutical company. We address rare, orphan diseases that typically have no available treatment, and strive to develop novel therapies which can have a positive impact on the lives of patients and families. As such, the needs of the patients who suffer from neglected rare diseases drive our mission first and foremost. Our goal is to provide rare disease patients with compelling new therapeutic options that will allow them to lead the fullest lives possible. Such diseases have been frequently overlooked for drug development and remain “orphan” due to the assumption that they have a small individual footprint. We aim to change this outlook, engaging in community awareness, regulatory outreach, and most importantly, delivering novel treatments. By focusing on rare and orphan diseases, we hope to bring a much-needed spotlight to the area by leveraging our collective conviction that these efforts will change the world for the better.