Tagged with Rare Diseases

India Swati Jajodia, managing director of Swati Spentose discusses her commitment to tackling underserved diseases and the company’s approach to bringing pentosan polysulfate to global markets while working with Key Opinion Leaders and CMEs in raising awareness about interstitial cystitis (IC).    Could you please introduce Swati Spentose to our international…

Switzerland Jürgen Pohle provides both a fascinating and open account of the latest movements of Neovii; a company that possesses the ability to make decisions at the speed of a startup but also has a heritage dating back to the 1970s. After an outstanding career spanning 25 years with Big Pharma…

Canada Marc LePage, president and CEO of Genome Canada describes the early days of the organization and talks modern day plans, such as personalized diagnostics for rare diseases, international alliances and genomic data collection and sharing.   Marc, you helped pioneer the creation of Genome in 2000 and led Génome Québec…

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…

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 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…

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…

Switzerland Dr. Andrea Michael Meyer, general manager of Sanofi Genzyme Switzerland, reveals how the Swiss business has been experiencing year-on-year double-digit growth; how rare diseases was the foundation of Sanofi Genzyme´s business and remains a key part of their DNA; and how a focus on patient centricity remains at the core…