Tagged with Rare Disease

Poland Mirosław Zieliński, president of the Polish National Forum for the Therapy of rare diseases (ORPHAN), the representative voice for 35 Polish rare disease associations, discusses the implications of the introduction of a national rare disease plan and the need to improve rare disease diagnostics. Furthermore, he highlights the importance of…

Canada Clarissa Desjardins is the founder and CEO since 2011 of Canadian biotech success story, Clementia Pharmaceuticals. She explains the different milestones of her company and her priorities moving forward. She also gives her insights on the entrepreneurship environment in Canada and advice on how to make it even more compelling…

Japan Makoto Suematsu, president of the Japan Agency for Medical Research and Development (AMED), discusses AMED’s successful unification of Japan’s previously disparate systems for medical research funding; the strategic significance of the country’s strong footprint in regenerative medicine and rare diseases; and the organization’s internationalization strategy, propelled by the push to…

Austria Rudolf Widmann, CEO and founder of AOP Orphan, the first rare disease-specific European company, discusses the overriding goal to ensure rare disease patients are matched with the correct treatments and the importance of open dialogue with specialists and patient groups. Furthermore, he highlights the need to differentiate health technology assessments…

Austria Dr. Wolfgang Harrer, regional manager of Chiesi Central and Eastern Europe (CEE), discusses the challenges of managing the region’s diverse cultural differences and the importance of CEE within global operations. Furthermore, he points out the factors influencing the influx of innovative drugs into Austria, especially in rare diseases, and highlights…

Austria David Kasper, CEO of ARCHIMEDlife, an innovative Austrian company focused on novel laboratory diagnostics, discusses the innovative solution of a dried blood sample in daily routine diagnostics and novel assays as well as the intricacies of working in rare diseases. Furthermore, he provides an insight into the company’s legacy project…

Austria Dr. Wolfgang Schnitzel, general manager of Shire Austria, and Karl-Heinz Hofbauer, site lead Vienna and managing director for the Technical Operations business, discuss the huge impact of Shire’s 32 billion USD acquisition of Baxalta that has grown the workforce from a few Shire employees in Austria to 3,500. Furthermore, they…

Colombia Gianfranco Biliotti, recently appointed General Manager for Alexion Colombia, shares his insights on the particularities of operating in the ultra-rare disease segment, while highlighting the importance of managerial flexibility, the company’s unmatched patient-centricity and close ties with doctors, the role of education in improving patients’ lives and his strategic priorities…

Greece A strong proponent of the value of orphan drugs, Shire Greece’s Managing Director, Christos Dakas, shares his insights on the recent merger with Baxalta, the necessity of exempting orphan and haemophilia drugs from clawbacks to circumvent market challenges for critical treatments, as well as his vision for the bright future…

Ireland James Lennertz, Group VP and GM EUMEA for BioMarin, discusses the key highlights of the past few years for BioMarin, their unique positioning as a company offering innovative therapies for ultra-rare diseases, the main challenge he sees for BioMarin’s international operations, and his priorities for the next few years. James,…

Switzerland Specializing in ultra-rare diseases, Alexion targets patient populations of less than 20 patients per million. Christophe Bourdon discusses Alexion’s progress towards developing access for patients across the EMEA region.   Since day one, Alexion’s mission has been to focus solely on discovering treatments for patients affected by these incredibly rare…

Mexico Leonardo Torres, general manager Mexico, Central America & Caribbean of Shire, provides insights into the rapidly progressing integration of Baxalta in Mexico, while he also highlights his key priorities to further accelerate the growth and consolidate the strategic positioning of Shire, a company that now proudly stands out as the…