Amicus Therapeutics is a biopharmaceutical company developing next-generation treatments for a broad range of human genetic diseases, with a focus on improving therapies for lysosomal storage diseases.
Alexion Pharmaceuticals, Inc.
Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is developing its next product, asfotase alfa, as an investigational treatment for patients with hypophosphatasia.
Batten Disease Support and Research Association
Batten Disease Support and Research Association: In order to effectively combat the devastation of the disease and to fully support research efforts to unravel the mysteries of Batten disease, the worlds of medical science, those affected by Batten disease and their families must meet and work closely together to reach understanding and common goals.
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc. BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.
The Fabry Support and Information Group
FSIG’s mission is to raise awareness of Fabry disease and its symptoms. FSIG’s primary goals are to educate and support. To help facilitate this FSIG maintains a confidential database of Fabry patients to assist in networking. This database also serves as a means of documenting the increasing numbers of diagnosed Fabry cases. FSIG also maintains a library of information about Fabry disease and is continually updating this body of knowledge.
Genzyme, a Sanofi company
Genzyme, a Sanofi company is committed to discovering and delivering transformative therapies for patients with rare and special unmet medical needs, providing hope where there was none before.
The Greenwood Genetic Center
The Greenwood Genetic Center is a nonprofit institute organized to provide clinical genetics services, diagnostic laboratory testing, educational programs and resources and research in the field of medical genetics.
At InsideMD, we are a survey tool that understands physicians. These surveys are created by doctors for doctors. After completing a survey, you can compare your responses with those peers to gain insights into what sets your practice apart from your colleagues.
The International Society for Mannosidosis and Related Diseases (ISMRD)
The International Society for Mannosidosis and Related Diseases (ISMRD) is an internationally focused not-for-proft organization whose mission is to advocate for families and patients affected by one of the following disorders: Alpha-Mannosidosis, Aspartylglucosaminuria, Beta-Mannosidosis, Fucosidosis, Galactosialidosis, Mucolipidosis I (Sialidosis), Mucolipidosis II alpha/beta (I-Cell Disease), Mucolipidosis III alpha/beta (Pseudo-Hurler Polydystrophy), Mucolipidosis III Gamma, and Schindler Disease.
LAL Aware is a non-profit organization who supports those who are affected by Lysosomal Acid Lipase Deficiency (LAL D). This group is made up of parents, patients and professionals directly affected by LAL D.
Lukare Medical, LLC
Lukare Medical, LLC is dedicated to ensuring the commercial viability of small market products that offer the highest value for patients with rare medical needs.
Lysosomal & Rare Disorders Research & Treatment Center
Internationally renowned experts focused on the individual patients with rare diseases, offering care by the highest standards, leading protocols and personalized services. We provide multi-center collaborative trials, investigator initiated studies, pilot and proof-of-concept studies and Bench-bedside protocols with expertise in translational medicine.
The MLD Foundation is a 501(c)(3) non-profit US tax-exempt organization formed in May 2001 to serve families throughout the world affected by metachromatic leukodystrophy (MLD), a terminal genetic disease.
Newborn Screening Translational Research Network (NBSTRN)
The Mission of the Newborn Screening Translational Research Network (NBSTRN) is to improve the health outcomes of newborns with genetic or congenital disorders by means of an infrastructure that allows investigators access to robust resources for newborn screening research.
Orsini Healthcare provides a unique combination of professional services, clinical teams, medical products, and a full-service pharmacy to transition from a health care facility to home or to manage a health condition. For complex medical conditions as well as routine health needs, Orsini delivers the guidance, confidence and expertise that is essential for successful outcomes.
Pfizer has a leading portfolio of products and medicines that support wellness and prevention, as well as treatment and cures for diseases across a broad range of therapeutic areas; and Pfizer has an industry-leading pipeline of promising new products that have the potential to confront some of the most challenging diseases of our time.
Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. is an emerging global biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases.
Rare Disease Report
Rare Disease Report is a website and weekly e-newsletter that offers an independent voice for the Rare Disease Community. It strives to bring together medical, scientific, investment, regulatory, and advocate professionals interested in rare diseases and orphan drugs. Rare Disease Report is proud to be the media partner for WORLDSymposium 2015.
Shire International focuses on developing treatments for conditions where the impact of medicine can make an immediate and tangible difference for patients. Shire provides treatments in Neuroscience, Rare Diseases, Gastrointestinal, and Internal Medicine.
Shire Pharmaceuticals is a global specialty biopharmaceutical company that works closely with specialist physicians, nurses, pharmacists and other healthcare professionals to develop and market medicines that aim to improve quality of life for patients, their families and carers.
Synageva BioPharma Corp. is a biopharmaceutical company with headquarters, research and development facilities in Lexington, MA. Their mission is to deliver breakthrough medicines globally for patients suffering from rare, devastating diseases. Their team has a proven track record of delivering life-altering therapies to patients with diseases once considered too rare for developing treatments.
Ultragenyx Pharmaceutical Inc.
Ultragenyx is a clinical-stage biotechnology company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious, debilitating genetic diseases.
University of Minnesota and Fairview Pharmacy Services
University of Minnesota Health, in partnership with Fairview Pharmacy Services, diagnoses and treats patients with rare diseases. Pharmacy services include comprehensive drug therapy management, oral and infused therapies, home infusion with nationwide care coordination and clinical trials services.