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Rare Disease Services In Usa
19 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: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: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 ...
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 ...
Manufactured by:RareStone Group based inShanghai, CHINA
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 rural regions and certain geographic regions. Liver disease accounts for approximately ...
Manufactured by:MorphoSys AG based inPlanegg, GERMANY
Expanded Access to our Investigational Medicinal Products Policy. At MorphoSys, our mission is to make innovative biopharmaceuticals to improve the lives of patients suffering from serious diseases. Before these and all other investigational medicinal products can be made broadly available, the U.S. Food and Drug Administration (FDA) and other health authorities around the world require that ...
by:Andelyn Biosciences based inColumbus, OHIO (USA)
Andelyn Biosciences provides clients with a broad range of viral vector manufacturing platforms and solutions, including AAV and Lentiviral adherent and suspension systems along with plasmid manufacturing. We support our clients throughout every step of cGMP manufacturing – from pre-clinical trials of gene-modified products and cell therapies to commercial ...
by:Avero Diagnostics based inIrving, TEXAS (USA)
Expectant parents want to do everything they can before the birth of their baby. The Avero Carrier Test looks at a patient’s genes to see if they may carry a disease that they could pass on to their child. Knowing a patient’s genetic carrier status is one of the most important things you can do to help them prepare for ...
Manufactured by:Pluristem Therapeutics Inc. based inHaifa, ISRAEL
Acute Radiation Syndrome (ARS) involves severe, potentially lethal damage to the bone marrow’s ability to produce blood cells, as well as to other systems and organs. Severe damage to bone marrow makes victims vulnerable to life-threatening hemorrhage, infection and anemia. ...
Manufactured by:Pluristem Therapeutics Inc. based inHaifa, ISRAEL
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 transplantation of multipotent hematopoietic cells, is a standard treatment in various malignant and non-malignant conditions. Transplant patients are ...