Rare Disease Services
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Manufactured by HemoShear Therapeutics, Inc.based in 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 ...
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Manufactured by RareStone Groupbased in 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 ...
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Manufactured by RareStone Groupbased in 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 ...
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based in FRANCE
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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Manufactured by RareStone Groupbased in 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, ...
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Manufactured by RareStone Groupbased in CHINA
per year worldwide, with 1 million due to complications of cirrhosis and 1 million due to viral hepatitis and hepatocellular carcinoma. Specific rare liver and kidney diseases often associated with metabolic syndrome as well as viral ...
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Manufactured by RareStone Groupbased in 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 ...
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Manufactured by CENTOGENE N.V.based in GERMANY
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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Manufactured by RareStone Groupbased in 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 ...
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based in USA
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 component. We are eager to discuss collaborative partnerships utilizing our proprietary ...
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Manufactured by BioMarinbased in 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 ...
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Manufactured by BioMarinbased in 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 ...
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based in GERMANY
Kearns-Sayre Syndrome (KSS) is a mitochondrial disease, with Rare Disease designation (7,400 – 22,000 patients in Europe), caused primarily by large-scale mitochondrial DNA deletions. Cardiac dysfunction is the principal clinical manifestation and primary cause of death. There are no current treatment options available that go ...
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Manufactured by CENTOGENE N.V.based in GERMANY
We provide a broad range of services to successfully operate clinical trials. 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 ...
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Manufactured by BioMarinbased in 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. ...
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based in SOUTH KOREA
the increase in NAD+, an essential cofactor of the redox reaction in cells. The goal is development of innovative new drug for metabolic diseases, rare diseases and refractory inflammatory ...
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Manufactured by Sangamo Therapeuticsbased in USA
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 ...
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based in CAMBODIA
enrollment to help you meet your recruitment goals. Find out how Verana Health can support your next rare disease, site-based, or multi-disciplinary ...
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by ICON plcbased in IRELAND
failure, lipid disorders, diabetes, renal insufficiency and renal transplant, arrhythmia, and rare ...
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Manufactured by Neovii Pharmaceuticals AGbased in SWITZERLAND
Aplastic anemia is a rare, but serious hematological disease caused by a failure or deficiency of the bone marrow to produce blood cells (erythrocytes, leukocytes, and platelets). Patients with aplastic anemia are at risk of life-threatening infections or fatal bleeding. Curative treatments for aplastic anemia include immunosuppressive ...
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