Tagged with Rare Disease

France Biogen France is operating at a moment of profound change, where rapid advances in neurology, immunology and rare diseases intersect with shifting global dynamics on investments to innovation and new expectations of industry-public collaboration. In this interview, Marina Vasiliou reflects on how these forces are reshaping the affiliate’s mission, the…

Tags: USA Manisha Balwani is Chief of the Division of Medical Genetics and Genomics at Icahn School of Medicine at Mount Sinai, where she leads programs spanning genetic screening, diagnosis, clinical trials, and treatment across the lifespan. In this interview, she discusses how Mount Sinai supports rare disease patients from early identification…

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…

USA Catherine Owen Adams, CEO of Acadia Pharmaceuticals, discusses how the company’s dual commercial pillars in Parkinson’s disease psychosis and Rett syndrome are delivering much-needed impact for patients with significant unmet needs and shaping Acadia’s next phase of growth. She shares insights into the company’s expansion plans, pipeline strategy, partnership goals,…

USA Michael Pearlmutter, CEO of the EveryLife Foundation for Rare Diseases (the EveryLife Foundation), brings a deeply personal motivation and commitment to driving meaningful change for the rare disease community. In this conversation, Pearlmutter reflects on the Foundation’s mission, its impact across the rare disease ecosystem, and the evolving opportunities to…

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…

USA Dr Jerry Vockley, Director of the Center for Rare Disease Therapy and Chief of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, offers a look at the evolving landscape of rare disease care across early diagnosis, newborn screening, natural history research, clinical trial design, and the persistent…

France Alexion France is entering a pivotal phase as the rare-disease landscape becomes more competitive, more data driven and increasingly shaped by early access and clinical-trial performance. In this interview, Celine Khalife reflects on France’s shifting position, the evolution of complement and science, the integration of Amolyt Pharma and the growing…

France Biocodex has evolved from a pioneering French laboratory built around a single yeast strain into an international healthcare group spanning microbiota, women’s health and a focused orphan-disease activity. In this conversation, CEO Nicolas Coudurier reflects on the company’s scientific roots, its expansion across more than one hundred markets, and the…