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Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Suppliers Serving Mongolia
43 companies found
based inHuningue, FRANCE
FIRALIS is a biotechnology company located in the very heart of BioValley, on the true triple border of FR-DE-CH, only a few minutes walking distance to Basel, the pharmaceutical capital of Europe. Firalis creates novel values via biomarker ...
based inBagsværd, DENMARK
We are a global healthcare company, founded in 1923 and headquartered just outside Copenhagen, Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity, and rare blood and rare endocrine diseases. ...
Drug development works best as a team endeavor. We continuously explore strategic collaborations to advance our pipeline toward commercialization and to further our work in the treatment of both rare diseases and common diseases that have a genetic ...
based inOxford, UNITED KINGDOM
Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on improving the natural delivery capabilities of exosomes, and developing an entirely new class of therapeutics. Backed by leading venture capital groups and ...
Niemann-Pick C disease (NPC) is a lysosomal storage disorder which affects the brain and multiple other ...
based inPhiladelphia, PENNSYLVANIA (USA)
We are a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare diseases. Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and ...
based inPrinceton, NEW JERSEY (USA)
The drug discovery and development process is long and expensive with more failures than successes. At Certara, we anticipate and address your critical drug discovery and development risks and decisions using biosimulation, technology and services. ...
based inBothell, WASHINGTON (USA)
AGC Biologics offers deep industry expertise and uniquely customized services for the scale-up and cGMP manufacture of protein-based therapeutics (using mammalian and microbial systems), mRNA, pDNA, viral vector and cell therapy products, from ...
based inPlanegg, GERMANY
TQ Therapeutics GmbH is pioneering a novel approach to cell and gene therapy with its TQx platform, which transforms the production of cellular therapies into a rapid, automated process that occurs at the patient's side. This method leverages ...
TQx is developing a modular target cell-specific modification platform including relevant technology components for the generation of gene modified cell therapy ...
based inCambridge, UNITED KINGDOM
We’re driven by the shared purpose of breaking new ground in the discovery of new treatments for rare diseases. Healx is a mission-driven technology company pioneering the next wave of drug discovery in order to bring novel, effective treatments to ...
Our Rare Treatment Accelerator helps academic, patient, and industry groups advance their rare disease drug redevelopment projects. By combining our team, expertise and financial resources with your research and insights, we can get treatments to ...
based inStockholm, SWEDEN
Gesynta Pharma is a Swedish pharmaceutical company that develops unique drug candidates with the aim to reduce harmful inflammation and increase blood flow in the body’s microvasculature, thereby providing safe and efficacious treatments for several ...
based inChatham, NEW JERSEY (USA)
Tonix is a clinical-stage biopharmaceutical company focused on developing novel therapies and vaccines to prevent and treat central nervous system disorders, immunology conditions, infectious diseases, and rare diseases. At Tonix, we are focused on ...
TNX-2900, Tonix’s proprietary potentiated intranasal oxytocin is in the pre-Investigational New Drug (IND) stage as a candidate for the treatment of Prader-Willi syndrome (PWS) and non-organic failure to thrive disease. TNX-2900 has been ...
based inGermantown, MARYLAND (USA)
Cell and gene therapies (CGTs) use modified cells or cellular matter to treat, prevent or cure serious health issues like cancer, genetic disorders, immunodeficiencies, rare diseases, and more. CGTs have shown potential to replace damaged cells with ...
based inShanghai, CHINA
RareStone Group is dedicated to building the first rare disease ecosystem in China to improve the lives of patients and families impacted by rare diseases, by bringing the best-in-the-world therapeutic solutions and comprehensive and sustainable ...
Chronic kidney disease (CKD) is a non-communicable disease usually caused by diabetes and hypertension. The prevalence of chronic kidney disease in China is reported to be 10.8%, and is predicted to increase in the next decade, especially among ...
based inNangang Distric, TAIWAN
We are a clinical-stage specialty pharmaceutical company dedicated to the development and commercialization of best-in-class novel nanomedicines that combine our proprietary lipid-assembled drug delivery platform with approved active pharmaceutical ...
based inAshiya, JAPAN
Since its establishment in 1975, JCR has been working on proprietary technology developments and creation of products in a manner to be 'one step ahead' of competitors at all times under its corporate philosophy, 'Contributing towards people's ...
based inSan Diego, CALIFORNIA (USA)
Rady Children’s Institute for Genomic Medicine is a non-profit research organization embedded within Rady Children’s Hospital-San Diego. We pioneered a medical revolution to end the diagnostic odyssey for neonatal and pediatric rare disease. Now ...
based inRostock, GERMANY
Since 2006, CENTOGENE has helped physicians diagnose thousands of rare disease patients around the world. A quick and accurate diagnosis can end an odyssey of medical tests and visits to multiple medical specialists. It also helps physicians better ...
Analyze, interpret, and report genomic variants with our decentralized diagnostic solution – no matter where you are located. CentoCloud is a cloud-based Software as a Service (SaaS) platform specifically designed for the rapid bioinformatic ...
based inBalerna, SWITZERLAND
APR Applied Pharma Research s.a. (APR) is a Swiss Pharma company dedicated to providing patients suffering from rare diseases with innovative treatments that make a real difference in their life. Our focus as a Swiss global health care company is ...
based inMilpitas, CALIFORNIA (USA)
ASC Therapeutics is a fully incorporated biopharmaceutical company focusing on the development of curative therapeutic products that are enabled by our proprietary gene editing platform, TARGATT and other gene editing technologies developed after 12 ...
based inBoston, MASSACHUSETTS (USA)
SOPHiA GENETICS is the creator of a global data-sharing network – we work with customers from over 790 institutions in over 70 countries. Our network advances data-driven medicine to improve health outcomes and economics worldwide. We offer a ...
Most rare and inherited diseases have a neurological component, likely because more than 80% of human genes are expressed in the brain.1 Neurological disorders are multifactorial and heterogeneous, meaning that their genetic basis is often poorly ...
based inLund, SWEDEN
Camurus is committed to developing and commercializing innovative and long-acting medicines for the treatment of severe and chronic conditions, including opioid dependence, pain, cancer and endocrine disorders. New drug products are based on our ...