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Rare Diseases Suppliers Serving Poland
69 companies found
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 inGhent/Zwijnaarde, BELGIUM
Confo Therapeutics’ unparalleled technology stabilizes functional conformations of GPCRs (G protein-coupled receptors) to uncover a wide range of previously inaccessible GPCRs as drug targets. This technology platform combined with the pharmacologic ...
based inStockholm, SWEDEN
IBT is a pharmaceutical company with its registered office in Stockholm with a vision to develop drugs influencing the human infant microbiome, and thereby prevent or treat rare diseases affecting premature infants. IBT is currently developing its ...
IBP-9414 for the prevention of necrotizing enterocolitis (NEC) contains the active substance Lactobacillus reuteri, which is a co-evolved human bacterial strain naturally present in breast milk. Lactobacillus reuteri is a live bacteria known to be ...
based inOxford, UNITED KINGDOM
As a leading global provider of diagnostic genomic solutions, our goal is to enable researchers and clinical decision makers to reach the right decisions for each patient, every time. The quality and innovation built into our products for NGS, ...
based inSan Francisco, CALIFORNIA (USA)
Invitae’s mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Like wearing sunscreen or getting your blood pressure checked at the doctor, genetic testing is another important ...
based inCarlton, AUSTRALIA
Radiopharm Theranostics has the ambition to become a recognised leader in the development of radiopharmaceutical products for both diagnostic and therapeutic uses in areas of high unmet medical needs. There is unique potential of ...
DUNP19 is the invention of Professor Ulmert at UCLA. ...
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 ...
We support programs and activities that foster excellence in patient care and provide valuable scientific, medical, and educational information to the medical and scientific communities. We also support clinicians and the independent research they ...
based inHaifa, ISRAEL
Pluristem Therapeutics Inc. is a clinical-stage regenerative medicine company using placental cells and a unique, proprietary, three-dimensional (3D) technology platform to develop cell therapies for conditions such as inflammation, muscle injuries, ...
Bone marrow deficiency refers to a condition in which the hematopoietic stem cells in the bone marrow fail to produce enough, or produce abnormal, red blood cells, white blood cells and platelets. Hematopoietic cell transplantation (HCT), the ...
based inVancouver, BRITISH COLUMBIA (CANADA)
Precision NanoSystems is a global leader in technologies, solutions and services for the development of non-viral delivery of genomic medicines, including mRNA vaccines and therapeutics. We work with the world’s leading drug developers to understand ...
based inNaarden, NETHERLANDS
Azafaros has leveraged on decades of discovery by professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands. Expanding on their research and led by highly experienced ...
Azafaros B.V. is pioneering the development of innovative oral small molecule compounds addressing the pathological effects of metabolite accumulation in rare metabolic disorders. Their lead compound, nizubaglustat, leverages unique ...
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 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 inSan Francisco, CAMBODIA
Verana Health is a digital health company that delivers quality drug lifecycle, medical practice, and medical society insights from an exclusive real-world data network of more than 20,000 healthcare providers. Our specialty medical society partners ...
From protocol development and clinical trial design to site selection and identifying potential participants*, we help speed patient enrollment to help you meet your recruitment goals. Find out how Verana Health can support your next ...
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 ...
based inMonmouth Junction, NEW JERSEY (USA)
MedChemExpress (MCE) offers a wide range of high-quality research chemicals and biochemicals (novel life-science reagents, reference compounds and natural compounds) for scientific use. We have professionally experienced and friendly staff to meet ...
Maralixibat (SHP625) chloride is an orally active ileal bile acid transporter (IBAT) inhibitor. Maralixibat chloride can be used for the research of rare cholestatic liver diseases including Alagille syndrome ...
based inMontreal, QUEBEC (CANADA)
SCIREQ strives to make a strong impact on global pulmonary health by empowering scientists to better understand and eradicate lung diseases. The global burden of respiratory diseases is primarily driven by asthma, chronic obstructive pulmonary ...
based inMontpellier, 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, ...
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 ...
based inLondon, UNITED KINGDOM
The Medical Algorithms Company supports providers and payers with analytics and documentation for evidence-based medical practice. Our platform supplies over 30,000 tested medical calculators and analytic risk scores. Nearly all specialties are ...
based inheongju-si, SOUTH KOREA
Curome Biosciences is an innovative R&D company developing therapies to treat mitochondrial dysfunction-related diseases with high unmet medical needs. Curome Biosciences wants to provide solutions for treatment of diseases with high unmet medical ...
Mitochondria are an essential cellular organelle that is responsible for the generation of energy (known as the ‘powerhouse of the cell’). Mitochondrial dysfunction is related with numerous diseases. Curome Biosciences ...
based inGangnam-gu, SOUTH KOREA
MitoImmune Therapeutics Inc. is a drug discovery and development biotech company that harnesses the power of mitochondria and its proprietary technology targeting mitochondria as the source for developing new potential breakthrough therapeutics. Our ...