Tagged with Rare Diseases

UAE With a relatively high number of genetic disorders, the Arab population of the United Arab Emirates (UAE) is increasingly becoming the subject of new studies. The database from the Center for Arab Genomics Studies indicates the presence of 241 genetic disorders among that demographic, a number they call “extremely high…

Opinion Monica Weldon, CEO and president of Bridge the Gap – SYNGAP Education and Research Foundation, shares her recent experience at the US Drug Law and Regulation course in Washington, DC and expands on the role that patient associations can play in the drug development process.   In today’s environment of…

UAE Khaled Elrefae, Middle East and Africa Specialty Care Operations Head (GM) for Ipsen, highlights Ipsen’s impressive internationalization strategy and the success factors as well as rationale behind the company entrance to the MEA market with the newly established affiliate in UAE. He then discusses his mandate to drive a transformation…

UAE The United Arab Emirates Genetic Diseases Association (UAEGDA) was established in 2006 and aims to increase awareness programs in order to prevent the occurrence of common genetic disorders in the UAE and throughout the region. Dr. Maryam Matar is the founder of the UAEGDA, and she discusses the importance of…

UAE The UAE ranks as a world leader in rapid drug approvals with its fast-track registration system that accelerates the process and eliminates long waiting times for innovative and orphan drugs.   If you bring innovation, the [UAE] government will support you on all levels Georg Schroeckenfuchs, Novartis In January 2018…

China Eric Bouteiller outlines the paradigm shift occurring in China around rare diseases and how the country has the potential to lead the global pharmaceutical industry in this vital area.   Through exchanges with public officials, academics, representatives of patient associations and industry experts, we sensed a paradigm shift The 8th…

Sweden Grifols is a pioneer in plasma science that develops plasma into essential medicines used to treat rare, chronic and life-threatening conditions. Eric Björnestål, general manager for the Nordic region, explains how the company is leveraging its knowledge of the public tender process to continue to grow in the region, highlights…

Sweden Olav Fromm, managing director of Chiesi Nordics, explains the deep roots of Chiesi in Sweden, where its blockbuster drug, a surfactant for treating neonatal babies, was developed. He also touches upon the strategic importance of the region to Chiesi, with the recent creation of a fully-fledged Nordic subsidiary, which will…

Spain Though still one of the younger and lesser-known outfits inhabiting the European drug development landscape, Irish-headquartered rare disease specialist, Jazz pharma has been making tremendous headway in the onco-haematology and sleep disorder spaces and the Spanish affiliate is absolutely no exception.   We blend the lessons of art with our…

USA PhRMA’s Anne McDonald Pritchett PhD highlights the remarkable development of cell and gene therapies in recent years and the three ways in which the US healthcare system can better accommodate them: creating manufacturing systems that take treatment timelines into account, a health system that values treatments based on patient outcomes,…

Taiwan Dr Hardy Chan shares the story of how he came out of retirement to lead Allianz Pharmascience; an up-and-coming biotech which aims to meet the unmet medical needs of rare disease patients, particularly in Asia. Dr Chan delves deep into the innovative science behind the company’s pipeline and shares the…

Sweden Amicus Therapeutics is a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases. Mattias Bankel, Head of Nordics and Baltics, shares his journey of establishing and developing Amicus’s presence in Sweden and other Nordic countries, furthermore his commitment to ensure that…