Fondation Ipsen Press

Every rare disease begins with a patient and a question: why isn't there a treatment? Too often, the answer isn't scientific, it is financial. The science exists. The will does exist. But the funding pathways don't. For decades, rare disease research has been caught between two worlds: public grants that are too short-term to sustain discovery, and private investment that sees too little market potential to engage. Thousands of promising ideas never leave the lab, not because they failed, but because the funding system did. This book is written to change that. Across the world, funders, patient organizations, and scientists are experimenting with bold, hybrid approaches that blend the discipline of finance with the purpose of medicine. Venture philanthropy, public-private partnerships, social impact bonds, and collaborative consortia are not financial curiosities, they are survival strategies for rare disease innovation. The aim of "Alternate Funding Mechanisms in Rare Diseases" is simple: to make these approaches understandable, usable, and actionable. Whether you lead a foundation, manage a research program, or simply care deeply about someone living with a rare condition, this book shows how to turn financial creativity into measurable impact. ABOUT THE AUTHOR Dr. Levine, President Fondation Ipsen, has three decades of experience in the healthcare sector principally at Mayo Clinic. For the last 6 years he has been President of Fondation Ipsen, an international science foundation focused on biotech innovation in Rare Diseases. A physician and scientist, James has published more than 200 articles, six papers in Science and Nature plus articles in journals such as, the New England Journal of Medicine, Lancet and JAMA. He has written four books published in 19 languages in 37 countries. His business training, focused on entrepreneurship, was from Harvard Business School. With a background in wearable technologies, data gathering and data analytics, and with more than 100 patents and trademarks, Levine helped found 35 companies. He was the Innovator of the Year in the state of Minnesota, the World Trade Fair and NASA. Under the Mayo Clinic NEAT Trademarks, Levine's team delivered scalable health solutions to 72 US corporations. A great deal of James' work focusses on biotech development, scalable health solutions and business opportunities in underserved regions in the United States, France and low/middle countries such as the Democratic Republic of Congo, Cote d'Ivoire, Afghanistan, Jamaica, Asia, Kenya and India. Consulting to the President of the United States, the US State Department, US Army and internationally, James in 2018 was awarded the President's Medal for promoting social embeddedness. 300 million people live with rare diseases without effective cures. Sustainable solutions require the efficient use of capital to optimize biotechnology companies, maximize impact and minimize suffering.

Living with a rare disease is not only a medical experience-it is a human one. It reaches into every part of life: the body that struggles, the mind that questions, the family that hopes, and the community that rallies. This book began with that truth and has followed it through the many ways people find comfort, meaning, and strength when medicine alone is not enough. Across these chapters we have explored how complementary practices-mindfulness, yoga, art, touch, and reflection-can restore balance and dignity. We have met caregivers who hold entire households together, and patients who, despite pain and uncertainty, continue to build lives that are creative, purposeful, and full of courage. The message is simple but profound: well-being is not a luxury or an afterthought. It is part of treatment itself. Every breath of calm, every shared laugh, every moment of understanding between a patient and a caregiver is an act of healing. ABOUT THE AUTHOR A graduate of Cambridge, James A. Levine became a physician by vocation and a writer almost by accident. He is a professor of medicine at the renowned Mayo Clinic in Minnesota, where he lives with his wife and their daughters. It was during a visit to the repugnant Rue des Cages in the Komatipura district of Bombay-where, under dramatic conditions, some of the 1.2 million children prostituted in India are forced to work-that James A. Levine saw a fifteen-year-old girl in a pink sari writing in a blue notebook. From this encounter came "The Blue Notebook", a powerful and unsettling first novel about child prostitution, the new slaves of the twenty-first century. The royalties from sales of The Blue Notebook are donated to the International Centre for Missing and Exploited Children. It was while traveling through emerging countries on behalf of the United Nations that the idea came to him to bear witness to the conditions in which his future heroes grow up and live. If you want a more literary tone-or a tighter, back-cover version-I can tweak the style.

The journal Science (American Association for the Advancement of Science) in collaboration with Fondation Ipsen delivers international science webinars for the general public. In 2024, these webinars focused on challenges in rare diseases. The Rare Disease Gazette is a magazine that broadcasts these discussions.

DACMAR is a FAST BUSINESS GUIDE for newbie biotech entrepreneurs. GIVE ME 5 MINUTES: If you know nothing about modern biotech business lingo, grab this book and read the LEARNING BOXES. In 5 minutes, you'll know DACMAR - you'll know enough! If you WANT TO BUILD a SUCCESSFUL biotech start-up that delivers new medicines, read the DACMAR ADVANTAGE. Each chapter will take you 5 minutes. By the time you are finished in less than 2 hours, you'll have the DACMAR ADVANTGE. DACMAR is the basic business building blocks for a biotech start-up. At the conclusion of the DACMAR ADVANTAGE, I take you to the WIN! Your start-up flourishes: you are acquired or go public. Through your entrepreneurial genius, millions of patients will be saved. The DACMAR ADVANTAGE will help you win. ABOUT THE AUTHOR James A. Levine - Dr. Levine, President Fondation Ipsen, has three decades of experience in the healthcare sector principally at Mayo Clinic. For the last 6 years he has been President of Fondation Ipsen, an international science foundation focused on biotech innovation in Rare Diseases. A physician and scientist, James has published more than 200 articles, six papers in Science and Nature plus articles in journals such as, the New England Journal of Medicine, Lancet and JAMA. He has written four books published in 19 languages in 37 countries. His business training, focused on entrepreneurship, was from Harvard Business School. With a background in wearable technologies, data gathering and data analytics, and with more than 100 patents and trademarks, Levine helped found 35 companies. He was the Innovator of the Year in the state of Minnesota, the World Trade Fair and NASA. Under the Mayo Clinic NEAT Trademarks, Levine's team delivered scalable health solutions to 72 US corporations. A great deal of James' work focusses on biotech development, scalable health solutions and business opportunities in underserved regions in the United States, France and low/middle countries such as the Democratic Republic of Congo, Cote d'Ivoire, Afghanistan, Jamaica, Asia, Kenya and India. Consulting to the President of the United States, the US State Department, US Army and internationally, James in 2018 was awarded the President's Medal for promoting social embeddedness. 300 million people live with rare diseases without effective cures. Sustainable solutions require the efficient use of capital to optimize biotechnology companies, maximize impact and minimize suffering.

Philanthropy is valued in French culture. According to the Fondation de France's barometer of French philanthropy, there are 4,650 Funds and Foundations in France in 2023. These structures commit nearly 11 billion euros per year to serve the general interest, an amount that continues to increase every year. Today, more than 30 billion euros in assets are held by these Funds and Foundations. Despite the deep-rooted value of philanthropy in French society, the non-for-profit sector is notoriously complicated in France. This book examines several types of foundations in France, including endowment funds, sheltered foundations, foundations recognized as being of public utility, and corporate foundations. Pros and cons for each are discussed in relation to their mission scope. Overall, understanding these distinctions is important for effective philanthropic strategies and for maximizing societal impact.

Les femmes sont sous-représentées dans les carrières en sciences, technologie, ingénierie, mathématiques et médecine (STEMM) et de nombreuses histoires personnelles témoignent des effets néfastes des préjugés, du harcèlement et de la discrimination dans le milieu scientifique. Malgré des progrès, il reste encore beaucoup à faire pour améliorer le recrutement, la pérennité et l'avancement des femmes dans les STEMM.Pratiques prometteuses pour remédier à la sous-représentation des femmes dans les domaines des sciences, de l'ingénierie et de la médecine est une approche intentionnelle et fondée sur des preuves visant à mettre en place des programmes, des interventions et des politiques qui peuvent rapidement avoir un impact positif dans les organisations académiques. Le changement positif est possible. Ce rapport, basé sur des données probantes collectées par des autorités mondiales, propose des solutions concrètes pour améliorer les opportunités de carrière des femmes dans les STEMM. À PROPOS DE L'AUTEURLes rapports d'étude consensuels publiés par les National Academies of Sciences, Engineering, and Medicine exposent le consensus fondé sur des preuves d'un comité d'auteures et auteurs ayant une expertise reconnue sur l'énoncé des tâches de l'étude. Les rapports contiennent généralement des résultats, conclusions et recommandations qui se fondent sur les informations recueillies par le Comité et qui résultent des délibérations du Comité. Chaque rapport a été soumis à l'examen rigoureux et indépendant de confrères et est représentatif de la position des National Academies sur la déclaration de mission. Les trois académies travaillent conjointement en tant que National Academies of Sciences, Engineering, and Medicine afin de fournir des analyses et des conseils indépendants et objectifs à la nation et de mener d'autres activités pour résoudre des problèmes complexes et éclairer les décisions de politique publique. Les National Academies encouragent également l'éducation et la recherche, reconnaissent les contributions exceptionnelles à l'enrichissement des connaissances et améliorent la compréhension du public en matière de science, d'ingénierie et de médecine.