Tagged with Rare Disease

Korea Brad Edwards became the first foreign general manager in the Lundbeck Korea’s 23-year history last year, joining from a background at Shire and Takeda across Australia and Singapore. He spoke to us about steering the company’s shift from psychiatry toward neuro-rare disease, the upcoming launch of its new migraine treatment,…

Germany DACH should become a powerhouse for the Group Germany is emerging as a key growth and launch market for Ipsen, with the DACH region now ranking as the Group’s leading European affiliate and gaining momentum across Oncology, Rare Disease and Neuroscience. As Ipsen enters its next phase of growth, new…

Europe Eight years into its shift into rare diseases, Ferrer is moving into a new phase as its pipeline matures. CEO Mario Rovirosa reflects on the setbacks and lessons shaping that journey, as well as the growing pressures on Europe’s ability to sustain innovation and patient access. Throughout, Ferrer’s commitment to…

Germany We have the agility to move quickly, the expertise to navigate complex healthcare systems, and the commitment to bring innovative medicines to patients who are often overlooked Norgine is reshaping its DACH business around a new growth engine, pairing a well-established portfolio with an expanded push into rare and ultra-rare…

Europe Europe accounts for a substantial share of Takeda’s business, hosts a significant slice of the company’s manufacturing footprint, and is involved in around half of its late-stage trials and priority development programmes. As the group prepares potential launches in narcolepsy, psoriasis and polycythaemia vera, a rare blood cancer, Gamze Yuceland explains how patient insight,…

Germany Growth in rare diseases is measured differently. It is not simply about launching new products, but about improving diagnosis, strengthening healthcare partnerships, and supporting patients throughout their journey. Those principles are shaping Orphalan’s next chapter, as the company expands beyond its longstanding focus on Wilson disease into new rare diseases.…

Germany The opportunity to continue growing the organisation, while driving performance and innovation, remains a strong source of motivation Sobi’s rapid growth in Germany and across DACH-NL is opening a new chapter for the rare disease specialist, as an expanding portfolio, new therapeutic areas and a growing organisation reshape the business.…

Germany Mirjam Mann, Managing Director of the German Alliance for Chronic Rare Diseases (ACHSE), discusses Germany’s progress and persistent gaps in rare disease care. She highlights a challenging landscape, with fragmented coordination, inadequate digital infrastructure, unsustainable registry funding, and a high burden placed on patients. Mann advocates for well-described and digital…

Europe The rare disease landscape is becoming increasingly sophisticated, demanding not only scientific innovation but also new approaches to access, evidence generation and patient support. Recordati Rare Diseases has built its growth by combining targeted business development with a lean, entrepreneurial organisation that remains closely connected to local healthcare systems. Luigi…

Germany RNA interference (RNAi) therapeutics have the potential to redefine how certain diseases may be treated in the future, shifting the focus from managing symptoms to addressing their underlying biological cause. Having led the way in translating RNA interference from a scientific discovery into an established class of medicines, Alnylam Pharmaceuticals…

Germany Dr Holm Graessner has spent more than 20 years building the infrastructure that rare disease patients in Europe often lack entirely: diagnostic pathways, European reference networks, and academic drug development for conditions too rare to interest any pharmaceutical company. As Managing Director of the Rare Disease Centre at Tübingen University…

USA Innovation cannot stop at the laboratory door. It must extend to how therapies are developed, approved, and reimbursed Brian Campbell, Vice President and General Manager of the US for Orchard Therapeutics, A Kyowa Kirin Company, discusses the critical role of newborn screening in identifying patients with metachromatic leukodystrophy (MLD), a…