Giuseppe Chiericatti of Chiesi Spain talks strategy, the development of the affiliate and shifting attitudes towards rare diseases in Spain.…

PJ Brooks PhD, program director in the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), National…

125 medicines for rare diseases were approved by the EMA between 2004 and 2014, of which 71 were granted orphan drug status. This chart shows the…

Levent Canyurt, CEO and founder of BIEM Ilac, shares how he transformed the company from a domestic pharmaceutical distributor into an international…

Abidin Gulmus, founder and chairman of Gen Ilaç, describes the company’s impressive journey to become Turkey’s principle orphan drug distributor…

Sea cucumbers – odd-looking marine animals with leathery skins and elongated bodies – can be found on ocean floors worldwide. Although more famous…

Korean Pharma company, Hanmi has won orphan drug status this week from the US Food and Drug Administration (FDA) for HM43239, a drug for the treatment…

Patient groups oftentimes serve as the authority on a specific disease when it comes to advancing research. “Without patient perspective,…

David Peña Castillo is the President and founder of The Mexican Federation of Rare Diseases (FEMEXER). Here he discusses what makes a patients…

After her son was diagnosed with SYNGAP1 - an extremely rare gene mutation - Monica founded Bridge the Gap – SYNGAP Education and Research…

Genethon, a laboratory created by the patient organization AFM-Telethon, is one of the world’s leaders in the development of biotherapies for rare…

Two months into the role, Umut Meriç, general manager for Chiesi Turkey, discusses the affiliate’s rapid progression and his plans to ensure…