NOVARTIS
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide.
Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram.
PTC BIO
PTC is committed to providing access to best-in-class treatments for patients with little to no treatment options. This mission underpins our focus on the discovery, development, and global commercialization of clinically differentiated medicines for those with rare diseases.
As a science-driven, global pharmaceutical company, we combine clinical and scientific expertise with groundbreaking science and technology. We work hard to provide broad access to our life-changing treatments. What drives us is our passion to provide patients with more moments with their families.
For over two decades, we have achieved historic breakthroughs in the treatment of rare diseases. We have been a pioneer in the development of treatments for rare diseases and we will continue to generate life-changing treatments around the globe for the next 25 years and beyond.
RECORDATI RARE DISEASES
At Recordati Rare Diseases, we focus on the few - those affected by rare diseases. We believe that every single patient has the right to the best possible treatment. They are at the core of our thinking and our actions. Recordati Rare Diseases is a pharmaceutical company that provides treatment for patients with rare diseases. Created in 1990, Recordati Rare Diseases is one of the most active companies in the field of rare diseases. Our specialty rare disease products are marketed directly by Recordati Rare Diseases in Europe, the Middle East, the U.S.A., Canada, Russia, Japan and Australia, in some Latin American countries, and through selected partners in other parts of the world.
Highly trained specialists and a scientific support team are available to collaborate with doctors, healthcare professionals, patients groups and families, to help improve the quality of life for people with rare diseases. Thanks to a unique production, packaging and distribution system developed for our products, our dedicated specialists are able to support patients in their home countries.
ROCHE
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics, as well as growing capabilities in the area of data-driven medical insights help Roche deliver truly personalised healthcare. Roche is working with partners across the healthcare sector to provide the best care for each person.
Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. In recent years, Roche has invested in genomic profiling and real-world data partnerships and has become an industry-leading partner for medical insights.
ASTRAZENECA
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide.
BIOMARIN
At BioMarin, we focus on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. BioMarin remains steadfast to its original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options, and are usually ignored.
As a biopharmaceutical company, our success stems from our focus on science and the hope our therapies can bring to patients. We are world leaders in metabolic disease innovation with multiple products commercialized and a growing pipeline of product candidates to address unmet medical needs. We continue to fuel our R&D engine by looking for opportunities that align with our strengths and competencies. And we relentlessly pursue exciting, early-stage science that has the potential to change the course of disease.
CHIESI GLOBAL RARE DISEASES
A Family owned and -run Certified B Corporation committed to Rare disease. At Chiesi, we are making a rare difference. We strive to create a world where it is common to have a therapy for all diseases and we act as a force for good, for society and the planet.
SANOFI
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
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