Rare Disease Manufacturers, Suppliers & Companies
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Manufacturerbased in Shanghai, 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 ...
RareStone - Online Rare Disease Consulting Services
With Zircon Internet+ Hospital, whose scope covers online medical consultation, remote diagnosis and treatment, AI medicine consultation, Doctors are able to make diagnosis and medical advices through on-line image text and image communication or ...
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Manufacturerbased in Charlottesville, VIRGINIA (USA)
HemoShear Therapeutics is a clinical stage company developing novel treatments for patients with rare diseases. We are implementing a new vision for how advanced biological and computational models can accelerate the discovery of innovative ...
Developing Treatments for Rare Diseases
Using our powerful REVEAL-TxTM drug discovery platform, our scientists are identifying and validating promising approaches to develop novel treatments aiming to extend and improve the quality of life of patients with rare ...
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Technologybased in Cambridge, 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 ...
Healx - Model Healnet - AI Platform for Rare Disease Drug Discovery
The world’s most efficient AI platform for rare disease drug discovery, supporting our teams and partners in our mission to advance new treatments towards the ...
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Software vendorbased in Boston, 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 ...
SOPHiA - Version DDM - Coud-Based Software Platform for Rare Diseases
Next-generation sequencing (NGS) of the human exome has the potential to identify pathogenic variants responsible for complex phenotypes associated with rare ...
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Manufacturerbased in Madison, WISCONSIN (USA)
InvivoSciences, Inc. (IVS) is a precision medicine company personalizing the drug development process by introducing patient-derived and gene-edited human samples, including 3D engineered microtissues. Our unique integration of cell & tissue ...
InvivioSciences - Rare Disease Drug Discovery Human Micro Tissue Technology
InvivoSciences (IVS) combines its human micro tissue technology and phenotypic assay platform with the systems biology approach to achieve rare disease modeling for its drug discovery. Our strip tissues are perfect ...
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Service providerbased in Bangalore, INDIA
Strand Life Sciences is a genomics-based research and diagnostics company with a mission to improve global health by decoding the genetic information contained in an individual’s genome. Strand Life Sciences partnership programs enable large-scale ...
SmartLab Services
Strand has extensive experience in validating NGS-based panels under CAP- and CLIA-settings: be it germline panels, somatic tumor panels, or liquid biopsy panels, and be it small panels with tens of genes, to large panels with thousands of ...
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Manufacturerbased in Rostock, 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 ...
Drug Discovery R&D Collaborations
the prevalence of a rare disease to disease modeling, we are your partner in de-risking drug development for rare diseases. ...
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Technologybased in Boston, MASSACHUSETTS (USA)
People living with rare and devastating diseases are our Guiding Star. We believe it is our responsibility to listen to, understand, and change the lives of patients and those who work tirelessly to help them. Our mission is to transform the lives ...
Alexio - Model RARE ANSWERS - System of Innovative and Sustainable Diagnostic Tools
People with rare diseases often wait years to receive a proper diagnosis and many never receive one at all. RARE ANSWERS™ is a system of innovative and sustainable tools designed to help ...
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Manufacturerbased in San 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 ...
Independent Research Services
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 ...
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Technologybased in Lexington, MASSACHUSETTS (USA)
At Dicerna, we create medicines that selectively silence the genes that cause disease using RNA interference, or RNAi. We are leaders in RNAi, harnessing the potential of this powerful and complex process to create medicines that can enhance the ...
Collaborating Services
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 ...
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Technologybased in Saint Kevin`s, IRELAND
At Horizon, we believe science and compassion must work together to transform lives. Our mission to deliver medicines for rare, autoimmune and severe inflammatory diseases and provide compassionate support comes from our strong and simple philosophy ...
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Manufacturerbased in New 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 ...
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Manufacturerbased in Philadelphia, 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 ...
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Technologybased in Wayne, PENNSYLVANIA (USA)
Palvella Therapeutics was founded to serve individuals suffering from rare diseases by developing safe and efficacious therapies that will dramatically enhance their quality of life. We focus on developing therapies that specifically target the root ...
Palvella - Model QTORIN - Microcystic LM
QTORIN™ rapamycin 3.9% is a novel targeted topical therapy being studied for Microcystic Lymphatic Malformations (Micro LM). Palvella is enrolling a phase 2 clinical study for people with Micro ...
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Manufacturerbased in Austin, 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 ...
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Software vendorbased in San Mateo, CALIFORNIA (USA)
NuMedii has been pioneering the use of Big Data, Artificial Intelligence (AI) and systems biology since 2010 to accelerate the discovery of precision therapies to address high unmet medical needs. NuMedii has active R&D collaborations with several ...
NuMedii - Artificial Intelligence (AI) Technology
NuMedii has been pioneering the use of Big Data, Artificial Intelligence (AI) and systems biology since 2010 to accelerate the discovery of precision therapies to address high unmet medical needs. ...
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Service providerbased in Wroclaw, POLAND
Pure Biologics is a biopharmaceutical company focused on the biological drug and non-systemic therapies discovery and development. We operate in the areas of immuno-oncology, autoimmunology, and rare neurological diseases, conducting research based ...
Pure Biologics - Model PB005 AptaMG - Therapeutic Medical Device
Myasthenia gravis (MG) is an autoimmune condition classified as a rare disease. Accordingly, myasthenic patients lack effective treatment options, as this rare disease is not a ...
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Service providerbased in Paris, FRANCE
Ipsen is a global biopharmaceutical group dedicated to improving lives and health outcomes through innovative medicines in Oncology, Neuroscience and Rare Disease. We also have a well-established and successful Consumer Healthcare business. We are ...
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Software vendorbased in Montpellier, FRANCE
SeqOne Genomics is a startup based in Montpellier in the South of France, founded in 2017. The company develops state-of-the-art genomics analysis tools for clinical applications in the fields of cancer and rare diseases. Its flagship product, ...
Rare and Hereditary Diseases Services
We offer a comprehensive solution for hereditary and rare disease data that detects both commonly accessible variants and relevant challenging variants such as mid-sized deletions, Alu elements and larger structural ...
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Manufacturerbased in Stockholm, 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 ...
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