Tagged with Rare Disease

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…

Spain Stefanie Granado, director general of Takeda Iberia recounts the opportunities available for Takeda in Spain following its acquisition of Shire. She also discusses Takeda’s local acquisition of TiGenix, handing Takeda stem cell production capabilities, and how this makes Spain a key market and hub for the company.   We continue…

USA Monica Weldon looks at the history of medicinal CBD, its current usages in the USA, and why robust scientific research on its efficacy need to be conducted in order to ensure the safety of rare disease patients – many of them children – who are using CBD products for their…

Spain Ines Perea, general manager for Spain and Portugal at Jazz Pharmaceuticals, a global biopharmaceutical company dedicated to developing life-changing medicines for people with limited or no options in the areas of sleep and hematology/oncology, discusses her progress over the last 14 months in nurturing the local affiliate and the importance…

France Franck Cousserans, general manager of France and Belgium at Jazz Pharmaceuticals, highlights the company’s key products and the challenges of market access in France. Furthermore, he indicates the steps needed to grow in the future and how Jazz’s corporate culture is key to its success.   In the US, Jazz…

UK Formed in 2015, listed UK start-up Amryt targets rare diseases with high unmet medical need. With one commercial asset on the market, a strong pipeline of development assets and a commercial infrastructure across the EMEA region already in place, the company is aiming to expand rapidly.   We have expanded…

Spain Aurora Berra, general manager of Sobi Spain and Portugal, highlights her key priorities and the opportunities and challenges inherent in the fragmented Spanish healthcare system. Berra gives insight into the need for a bespoke rare disease focus and delivers advice to young women starting their journey in the healthcare industry.…

Opinion Rhenu Buller, an expert on disruptive technologies at business consultancy Frost & Sullivan’s Asia Pacific office, outlines the technological innovations currently driving the drug development pipelines of rare disease companies.   Recent advancements in the development of novel platforms for precise targeting of small molecules, gene therapy, stem cell therapy,…

Opinion Kevin Rufang Huang is the president and founder of the Chinese Organization for Rare Disorders (CORD). A rare disease patient himself, he is responsible for bringing“International Rare Disease Day” to China. Consequently, rare diseases now have much more visibility in the country. As an expert on the subject, Kevin breaks…

Opinion Drug development in rare diseases is currently in a very exciting time. While much of the focus is on the scientific advances,  access to rare disease patients and well-designed clinical trials are also essential to evaluating new therapeutics. In this article, PJ Brooks, Program Director at the Office of Rare…

Korea Min-Young Kim, general manager at Ipsen Korea discusses her strategy to refocus the Korean affiliate away from the toxin market and towards oncology and rare diseases. She also documents Ipsen’s rapid local growth and increasing market share. Miss Kim also gives her assessment of the current drug pricing situation, as…

Rare Diseases Long regarded as a neglected backwater within the drug discovery landscape and the preserve of only a handful of niche players, the rare disease space has been undergoing an extraordinary turnaround in fortunes of late.   According to EvaluatePharma’s forecasts, orphan drug sales are set to grow 11 percent each…