Aceragen is a rare and orphan disease biopharmaceutical company. Our goal is to provide rare-disease patients with compelling new therapeutic options. Acid ceramidase deficiency presenting as Farber disease represents the first example of our approach in action. The disease can result in a great deal of suffering for patients and families. Aceragen is engaged in the clinical development of a recombinant version of the acid ceramidase enzyme (ACG-801).