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Rare Disease Services Available In In Usa
28 services found
Manufactured by:HemoShear Therapeutics, Inc. based inCharlottesville, VIRGINIA (USA)
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 diseases. In our most advanced program, the company is conducting a phase 2 clinical trial of HST5040, an oral small molecule investigational ...
Manufactured by:RareStone Group based inShanghai, CHINA
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 live-talk, without going to the hospital ...
Manufactured by:CENTOGENE N.V. based inRostock, GERMANY
We work with partners to develop new disease models and new biomarkers of disease. We can also use our disease models to identify new drug targets and screen chemical compounds to identify potential new drug candidates. CENTOGENE provides companies with tools to accelerate drug discovery. From population screening studies that elucidate the ...
Manufactured by:RareStone Group based inShanghai, CHINA
We're building a rare disease ecosystem to bring both a support network and much-needed treatments to individuals and their families affected by rare disease. We are developing an integrated platform that can support the scalable commercialization of rare disease drugs in ...
Manufactured by:RareStone Group based inShanghai, CHINA
The endocrine and metabolic system includes endocrine glands and endocrine tissues, which regulate human function by secreting hormones. Endocrine diseases are rare and often very difficult to diagnose and treat. Among the 121 rare diseases included on China’s national list of rare diseases ...
Manufactured by:Novo Nordisk A/S based inBagsværd, DENMARK
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 component. We are eager to discuss collaborative partnerships utilizing our proprietary RNAi ...
Manufactured by:BioMarin based inSan Rafael, CALIFORNIA (USA)
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 ...
Manufactured by:BioMarin based inSan Rafael, CALIFORNIA (USA)
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 ...
Manufactured by:BioMarin based inSan Rafael, CALIFORNIA (USA)
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 conduct to advance knowledge about rare diseases and/or BioMarin’s therapies and products. We do not provide ...
Manufactured by:RareStone Group based inShanghai, CHINA
Rare neurological diseases include developmental defects of the nervous system, genetic and metabolic diseases, neuroimmune diseases, neurological tumors, special types of infectious diseases of the nervous system, neurodegenerative diseases, and neuromuscular diseases. At ...
Manufactured by:CENTOGENE N.V. based inRostock, GERMANY
We leverage our proprietary Bio/Databank, multiomic platform, extensive physician network and our experts to ensure efficient and effective solutions to drive progress in the rare disease ...
by:Curome BioSciences based inheongju-si, SOUTH KOREA
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 is researching a treatment to improve mitochondrial dysfunction by inducing mitochondrial activation through the increase in NAD+, an essential ...
Manufactured by:Sangamo Therapeutics based inBrisbane, CALIFORNIA (USA)
Behind these are research efforts thoughtfully focused on leveraging our differentiated zinc finger technology. We are advancing a robust pipeline of wholly owned and partnered programs in rare disease, neurology, oncology and autoimmune indications with the goal of developing genomic medicines that transform the lives of ...
by:Verana Health based inSan Francisco, CAMBODIA
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 rare disease, site-based, or multi-disciplinary ...
by:ICON plc based inDublin, IRELAND
We have over 25 years of experience conducting cardiovascular trials, with particular emphasis on key cardiovascular/metabolic indications: heart failure, lipid disorders, diabetes, renal insufficiency and renal transplant, arrhythmia, and rare ...
Manufactured by:CENTOGENE N.V. based inRostock, GERMANY
Whole Exome Sequencing (WES) is a comprehensive genetic test that identifies changes in a patient's DNA that are causative or related to their medical concerns. By focusing on the entire protein-coding regions of the genome – the exome – WES offers you the coverage you need to diagnose patients rapidly and ...
Manufactured by:RareStone Group based inShanghai, CHINA
In recent years, the incidence of childhood cancer has been on the rise, and malignant tumor has become one of the main causes of death among adolescents. More than 250,000 children worldwide are diagnosed with cancer each year, and about 90,000 children die of cancer—making it the leading cause of death among children. In addition to leukemia, malignant lymphoma and other blood tumors, ...
by:Nutcracker Therapeutics, Inc based inEmeryville, CALIFORNIA (USA)
RNA is often seen as a molecule with a supporting role in human physiology– a go-between for DNA and proteins. In fact, RNA is a macromolecule with immense versatility, which can be harnessed to tackle the most complex and difficult to treat ...
by:JADBio based inLos Angeles, CALIFORNIA (USA)
AutoML brings democratization to the data analysis process by allowing everyone to utilize Machine Learning. Fully automate your data analysis, end-to-end, with AI and our purpose-built algorithms starting from preprocessing to interpreting the final ...
by:Strand Life Sciences Pvt. Ltd. based inBangalore, INDIA
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 ...