Tagged with Rare Disease

Czech Republic Patrik Zachar, Vice President Central Europe of Ipsen, shares how the company has built a strong presence in oncology and neuroscience in the CE region and the Czech Republic, quickly establishing market-leading positions in the indications they compete in. Whilst in neuroscience, Zachar still sees significant growth potential for its…

UAE Genpharm is a Dubai-based regional company providing market access and marketing solutions for rare disease products in the MENA region. Co-Founders Karim Smaira and Kamel Ghammachi provide insights into the rare disease space in the region, explain how the company is positioning itself in the gene therapy arena and why…

France These are worrying times for iconic French mid-cap Ipsen. Trials for palovarotene, a treatment for rare bone disorders, have been plagued by setbacks, perhaps contributing to erstwhile CEO David Meek’s decision to quit the firm in December 2019. The company’s share price has since plummeted.   Meek’s resignation to join…

Hungary Tamás Demeter, Country Manager for AOP Orphan shares his insights on the footprint of the company in Hungary, the areas of therapeutic need that AOP caters to, the performance of 2019 and its key achievements, the ecosystem of rare diseases in Hungary, and patient awareness while highlighting the role of…

UAE Myriam Hakim, the recently appointed GM for Japanese specialty pharma firm Kyowa Kirin’s UAE affiliate, discusses the strategic significance of the UAE and the Gulf region as a whole to the global group.   I appreciate all the efforts the [UAE] authorities have been putting into fast-tracking registration for innovative…

Canada Pamela Fralick of Innovative Medicines Canada (IMC) advocates for a collaborative multi-stakeholder approach to better tackle rare diseases in Canada.   Especially in smaller provinces, it’s virtually impossible to predictably manage the affordability and accessibility of drugs necessary to treat rare disease Around the world, rare diseases are becoming easier…

Hungary Laszlo Dinca, managing director of Vanessa Research – Hungary, shares his insights on the company’s rare and orphan disease portfolio; the intentions behind the acquisition of Hungaro-Gal Pharmaceutical Manufacturing [HG Biotech]; Hungary as a development market for biotechnology companies; and the future objectives of the company.   It is paramount…

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…