Areas of Research

Rare Diseases
Sigma-Tau Pharmaceuticals, Inc., a wholly owned U.S. subsidiary of Sigma-Tau S.p.A., is particularly proud of its research in the area of rare diseases. A rare disease is defined as any disease or condition which affects less than 200,000 persons in the United States. Sigma-Tau’s core belief and dedication is that the study of rare diseases should be an integral part of our research activity. This dates back many years.

Sigma-Tau has always believed that research in molecular biology would bring about the subdivision of diseases into smaller subgroups, and that the experience accumulated in the field of rare diseases would be essential to the development of a new generation of pharmaceuticals. This endeavor is underscored by the fact that in 1984 Sigma-Tau became the fourth company in the world to obtain an Orphan Drug Designation in the United States. The company later obtained seven more.

In partnership with the National Organization for Rare Diseases (NORD), Sigma-Tau has heightened awareness of rare diseases in the medical community through the publication of the Physicians' Guide to Rare Diseases. This guide has received widespread recognition for its role in helping doctors recognize and diagnose some of the world’s rarest medical conditions.

For the Sigma-Tau group, one of the primary reasons for conducting research is to explore entirely new approaches to treating disease. This is illustrated by the company's study of the biology underlying disease, an alternative approach referred to as "biologic pharmacology." The research is based on the assumption that disease begins at the metabolic level, when the biochemical pathways within cells and communication among them are altered.