Tagged with Rare Diseases

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…

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…