Tagged with Rare Disease

Hong Kong At Hong Kong Children’s Hospital (HKCH), a new chapter in rare disease medicine is unfolding. Under the leadership of Dr Luk Ho-ming, Chief of Service of the Department of Clinical Genetics, a multidisciplinary team is harnessing the power of genomics, data, and collaboration to bring answers to families who have…

Hong Kong Established in 2018 under the Hospital Authority, the Hong Kong Children’s Hospital (HKCH) has rapidly emerged as the city’s leading centre for complex paediatric and rare-disease care. Bringing together advanced diagnostics, pioneering research, and multidisciplinary expertise under one roof, HKCH represents a new model of integrated medicine in Asia, one…

France With France at the forefront of rare disease expertise yet increasingly challenged on patient access, Amicus Therapeutics finds itself at the heart of both progress and paradox. In this interview, Caroline Yau, General Manager for France & Benelux, discusses her transition from Big Pharma to biotech, the realities of operating…

France France has long been held up as Europe’s reference point for rare disease care – the first EU country with a national plan, a pioneer of coordinated expert networks, and home to some of the continent’s most influential patient organisations. More than three million French citizens live with one of…

USA Duane Barnes, President of Sobi North America, outlines how the company has rapidly expanded its presence in the region to become a key player in rare diseases, growing its US business more than twenty-fold over the past decade. He discusses Sobi’s strong focus on haematology and immunology, the company’s upcoming…

France From Hungary to Poland and now France, Nienke Feenstra has navigated some of Europe’s most complex healthcare landscapes. Today, as GM of Takeda France, she reflects on the country’s scientific strengths, its vital early access frameworks, and the evolving challenges of sustainability and digital transformation, always with patient value at…

France Francois LAMY, Vice President of AFM-Téléthon and father of a child with Duchenne muscular dystrophy, shares how the organisation grew from a family-led initiative into a major force in rare disease research and advocacy. He highlights AFM’s role in launching Genethon and advancing gene therapy, while criticising the lack of…

India As rare disease regulation moves from the margins to the mainstream of global health policy, India faces both a challenge and an opportunity: how to align with international best practices while designing solutions suited to its vast population and limited resources. This article — authored by experts from Power In…

France With only a handful of patients affected by each condition, rare diseases present a unique set of scientific, medical, and economic challenges. At the heart of this complex landscape is Recordati Rare Diseases, whose European headquarters and manufacturing footprint are anchored in France. General Manager Jean-Claude Roche shares his perspective…

Europe Affordability is not necessarily the main barrier to better rare disease therapy access in Europe. Across the continent, orphan availability correlates poorly with GDP per capita, but strongly with health technology assessment methods, speed of pricing and reimbursement, and central vs regional decision-making. Better system design – rather than simply…

Alexion Dana Vigier, Regional Vice President at Alexion, AstraZeneca Rare Disease blends clinical training as a French general practitioner with broad pharmaceutical leadership experience. Formerly President of the French Pharma Association in Oncology and a Global Franchise leader at AstraZeneca, she now oversees Alexion’s operations across Central and Northern Europe, including…

France Professor Bana Jabri, Director of the Institut Imagine, brings a transatlantic perspective to advancing genetic medicine. After 25 years in the US, she now leads efforts in France to harness genomic technologies, foster interdisciplinary collaboration, and modernise academic–industry partnerships. Her vision calls for strategic investment, institutional autonomy, and greater risk…