Program Supporter
BioMarin Pharmaceuticals
In 2018, BioMarin continued to pursue bold science and to work toward changing the practice of medicine in rare genetic diseases. We drove our business forward as we accomplished one product approval in the U.S. and reached several key milestones for two product candidates in Phase 3 of development. Scientifically, we seek to correct serious genetic diseases either by replacing what is missing or prompting the body itself to start providing what it needs. Our goal is to help the communities of people with rare diseases perform biologically the same as or similarly to people without disease.
Program Supporter
Nutricia North America
At Nutricia we believe in the power of nutrition to make a positive and proven difference to health. Everyday we apply our collective expertise in nutrition to some of the world's biggest health challenges in life in the nutritional management of diseases, disorders and medical conditions.
Exhibitor Sponsor
Acer Therapeutics
Acer Therapeutics develops therapies for patients with serious, rare and life-threatening diseases. Acer is committed to supporting patients and families who need it most. Acer is changing the path to managing serious rare and life-threatening diseases.
Exhibitor Sponsor
Aeglea Biotherapeutics
Aeglea was founded in 2013 to re-imagine the potential of human enzyme therapeutics. Our relentless mission is to deliver disruptive solutions that can provide meaningful benefit for many patients with rare genetic diseases. Every day, we strive to…
- Look beyond the conventional, redefining the potential of human enzymes to deliver disruptive solutions
- Work with purpose, driven by the urgent needs of the communities we serve
- Pursue our vision to become the premier human enzyme company
- Ultimately, change the lives of patients and their families now and for the future.
- Look beyond the conventional, redefining the potential of human enzymes to deliver disruptive solutions
- Work with purpose, driven by the urgent needs of the communities we serve
- Pursue our vision to become the premier human enzyme company
- Ultimately, change the lives of patients and their families now and for the future.
Exhibitor Sponsor
Cycle Pharma
Innovation. Transformation. Treatment, re-imagined. We re-imagine how drugs can benefit patients, to make their lives easier and improve their quality of life at every stage. By optimizing and enhancing treatments, improving efficacy, identifying new indications for drugs, and reintroducing products to the market, we give patients the options they need.
Exhibitor Sponsor
Eton Pharmaceuticals
Providing treatment for those in the greatest need. Together with our investors, without whom we could never achieve this mission, we take pride in helping rare disease patients overcome unthinkable challenges to live up to their greatest potential.
Exhibitor Sponsor
Horizon Therapeutics
At Horizon, we define our success by a different set of numbers: the number of lives touched, the number of lives changed, the number of lives saved. As we build a growing pipeline and explore all potential uses for our medicines, we strive to make a powerful difference for our patients, their caregivers and physicians every day. Our drive to deliver breakthrough therapies comes from our deep understanding of the patient journey. These patients’ courage inspires us – and drives us to listen, collaborate and build communities. That’s why, at our core, we believe science and compassion must work together to transform lives.
Exhibitor Sponsor
Recordati Rare Diseases
Recordati, established in 1926, is an international pharmaceutical group, listed on the Italian Stock Exchange (Reuters RECI.MI, Bloomberg REC IM, ISIN IT 0003828271), with a total staff of more than 4,100, dedicated to the research, development, manufacturing and marketing of pharmaceuticals. Headquartered in Milan, Italy, Recordati has operations throughout the whole of Europe, including Russia, Turkey, North Africa, the United States of America, Canada, Mexico, some South American countries, Japan and Australia. An efficient field force of medical representatives promotes a wide range of innovative pharmaceuticals, both proprietary and under license, in a number of therapeutic areas including a specialized business dedicated to treatments for rare diseases. Recordati is a partner of choice for new product licenses for its territories. Recordati is committed to the research and development of new specialties with a focus on treatments for rare diseases.
Exhibitor Sponsor
Ultragenyx Pharmaceutical
Ultragenyx is a bio-pharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are no approved therapies. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.