Tagged with Rare diseases

USA Charlene Son Rigby, CEO of Global Genes, shares her insights on the rapidly evolving rare disease landscape. She highlights exciting developments such as patient-driven research and development and the future potential of platform technologies and individualized medicines. Drawing from her personal experience, Charlene also emphasizes the critical importance of ongoing…

France Genethon sits at the forefront of a fast-accelerating moment in gene medicine, where decades of foundational research are now translating into clinical progress across rare liver, neuromuscular, immune and ophthalmic diseases. In this interview, Frédéric Revah reflects on the organisation’s scientific evolution, its approach to partnerships and access, and the…

France Norgine is redefining what a mid-sized European specialist can achieve, shifting from its gastroenterology heritage into a broader mix of consumer health, women’s health and rare diseases. These changes are taking shape across the organisation, with France playing an important role under Cyril Tavier’s leadership, from implementing new omnichannel models…

France Ipsen France is navigating a pivotal moment. The affiliate sits at the intersection of global pipeline expansion, rising expectations for access and renewed national ambition around industrial sovereignty and innovation. In this conversation, Dominique Bery explains how France is shaping Ipsen’s next phase of growth, from oncology leadership to deeper…

Brazil Rare diseases have long fallen through the cracks of Latin America’s health systems, but few figures have done more to change that reality than Toni Daher. What started as a father’s search for answers became Casa Hunter and, later, Casa dos Raros – a model of integrated, data-driven 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…