Refine by
Locations
- USA
- Alabama
- Alaska
- Arizona
- Arkansas
- California
- Colorado
- Connecticut
- Delaware
- District Of Columbia
- Florida
- Georgia (Us)
- Hawaii
- Idaho
- Illinois
- Indiana
- Iowa
- Kansas
- Kentucky
- Louisiana
- Maine
- Maryland
- Massachusetts
- Michigan
- Minnesota
- Mississippi
- Missouri
- Montana
- Nebraska
- Nevada
- New Hampshire
- New Jersey
- New Mexico
- New York
- North Carolina
- North Dakota
- Ohio
- Oklahoma
- Oregon
- Pennsylvania
- Rhode Island
- South Carolina
- South Dakota
- Tennessee
- Texas
- Utah
- Vermont
- Virginia
- Washington
- West Virginia
- Wisconsin
- Wyoming
Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Suppliers In Usa
37 companies found
based inNew York, NEW YORK (USA)
Protara is committed to identifying and advancing transformative therapies for people with cancer and rare diseases. The Protara team prioritizes creativity, diverse perspectives and tenacity to expedite our goal of bringing life-changing therapies ...
based inAustin, TEXAS (USA)
Lumos Pharma is a clinical-stage biopharmaceutical company whose mission is to develop new therapies for people with rare diseases, prioritizing its focus where the medical need is high, and the pathophysiology is clear. We are committed to this ...
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 inManassas, VIRGINIA (USA)
The Jeeva eClinical Cloud was developed by researchers with empathy who listen and learn to help clinical researchers, hospital sites, academic medical centers, CROs, and biopharmaceutical sponsors accelerate patient enrollment by 3x faster. Jeeva ...
based inWhite Plains, NEW YORK (USA)
Oligomerix is a biotechnology company focused on developing disease-modifying and treatment therapeutics for neurodegenerative diseases characterized by aberrant tau protein ranging from rare tauopathies such as progressive supranuclear palsy ...
There exist numerous rare diseases that tau dysfunction is associated with known as tauopathies. While Alzheimer’s is the largest tauopathy, other tauopathies are rare diseases that present accelerated regulatory pathways and significant ...
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 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 inMelville, NEW YORK (USA)
Population Bio, Inc. is a privately held precision medicine company that is empowering pharmaceutical companies to develop targeted therapies and companion diagnostics faster and more cost effectively. We are accelerating the delivery of precision ...
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 inTarrytown, NEW YORK (USA)
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently ...
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 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 inSan Rafael, CALIFORNIA (USA)
Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and ...
In alignment with our commitment to scientific innovation and areas of unmet medical need, we may provide product and funding support for independent research in rare diseases and genetic ...
based inNew York City, NEW YORK (USA)
Woolsey Pharmaceuticals is focused on the treatment of rare, neurodegenerative diseases. Many neurodegenerative diseases – including Alzheimer’s, vascular dementia, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, Huntington’s disease, ...
based inPlymouth Meeting, PENNSYLVANIA (USA)
Harmony Biosciences is a commercial-stage pharmaceutical company dedicated to developing and delivering innovative therapies for people living with rare neurological diseases, especially sleep/wake, neurobehavioral, and rare epilepsy disorders. The ...
based inRedwood City, CALIFORNIA (USA)
At Codexis, we apply our technology platform to engineer enzymes for biosolutions that benefit human health and environmental health. From developing biocatalysts for sustainable manufacturing of pharmaceuticals and food ingredients, to engineering ...
based inKing of Prussia, PENNSYLVANIA (USA)
Behring's parent company, CSL, was formed more than 100 years ago to save lives using the latest technologies. In the century since, CSL Behring has grown into a global biotechnology leader, driven by that same promise to save and improve lives. We ...
based inConshohocken, PENNSYLVANIA (USA)
BTG Specialty Pharmaceuticals provides rescue medicines typically used in emergency rooms and intensive care units to treat patients for whom there are limited treatment options. We are dedicated to delivering quality medicines that make a real ...
based inTorrance, CALIFORNIA (USA)
Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases. Our initial efforts have focused on treatments for ...
based inSan Carlos, CALIFORNIA (USA)
Developing live biotherapeutics to prevent and treat diseases of high unmet medical need. We leverage our deep knowledge of the human microbiome and its regulatory and developmental functions to develop live biotherapeutic products (LBPs) and ...