February 29^th is the World Day of rare diseases. In Italy there are around 2 million people suffering from this pathology. A rare disease is a pathology that affects limited number of people, precisely 1 out of 2,000. and for this reason, is difficult to identify and to treat. This means that many subjects don’t have a certain diagnosis and cure. The rare disease also directly affects the families and friends of the affected that need constant assistance and support.

The involvement of everyone is therefore necessary for providing cures, as happened with cystic fibrosis, the most widespread rare genetic disease in Europe that affect the respiratory apparatus.

PlumeStars, an innovative SME active at the Department of Food and Drug at the University of Parma, founded in 2017, contributed to studies on rare disease and precisely cystic fibrosis, telangiectasia, lung mesothelioma, glioma and GLUT1 diseases or cancers.

PlumeStars acquired the capability to develop novel orphan medicines based on pharmaceutical technologies of drug delivery applied to different drugs formulated in innovative medicines. The rare diseases are in large part genetic in origin and the cure consists in the development of DNA defect corrections. However, waiting for these DNA medicines the possibility to improve the quality of life with medicines based on innovative drug delivery is a strategy that can counteract the adverse effect of the genetic defect.

PlumeStars got the orphan designation of three medicines. Amikacin & Tobramycin powders for inhalations in cystic fibrosis, Cisplatin films for loco-regional delivery in pleura mesothelioma recurrences, Thalidomide powder for oral and nasal delivery in Hereditary Hemorrhagic Telangiectasia, are EMA and FDA designed orphan medicines assigned to PlumeStars.

Scientific research, in all fields nor only genetic can make possible to advance important steps in the treatment of many rare diseases. We strongly hope that, thanks also to the help and involvement of all doctors and researchers, there will no longer have to be a world day to remember the importance of every single patient.