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Rare Disease Product Applications In Usa
10 applications found
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 in 2018, more than 20 are endocrine/metabolic-related rare ...
ByRareStone Group based in Shanghai, CHINA
We are currently focusing on 4 medical areas - infectious diseases, immuno-oncology, rare diseases and autoimmune ...
ByModerna, Inc. based in Cambridge, MASSACHUSETTS (USA)
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 present, more than 7,000 rare diseases have been found in humans, about 60% of which are ...
ByRareStone Group based in Shanghai, CHINA
Thirdly, to achieve targeted treatment of rare diseases and tumors through research on small nucleic acid drugs. In addition, we can repair abnormal genes by performing gene editing at the DNA level to achieve targeted mutations of genes. The treatment of diseases by genetic methods are more targeted, safer and more effective than traditional ...
ByCD Biosciences - htDNA-chip® based in Shirley, NEW YORK (USA)
Our product pipeline shows the progress we’re making on clinical programs currently in development to create mRNA medicines for a wide range of diseases and conditions. We are proud of the advancements we’ve made in pioneering new mRNA vaccines and treatments that may have the potential to treat and prevent rare ...
ByModerna, Inc. based in Cambridge, MASSACHUSETTS (USA)
Lysosomal diseases are a group of rare, genetic metabolic diseases in which cellular waste products build up in the body. These disorders are generally progressive and there is no cure. Many lysosomal diseases have no FDA-approved ...
ByEli Lilly and Company based in Indianapolis, INDIANA (USA)
Maximize the probability of your clinical trial success by accelerating patient enrollment and flexible operations. Whether it is investigator-initiated study, observational study, phase 0, 1, 2, 3, or 4 post-market trials, real-world evidence generation to supplement your randomized controlled clinical trial, maximizing the speed of patient enrollment is the proven way to minimize your ...
ByJeeva Informatics Solutions Inc. based in Manassas, VIRGINIA (USA)
Save 70% of your time and maximize the probability of success of your clinical study. Avoid common pitfalls by starting your cohort study, patient registry, observational data collection, or clinical trial on Jeeva eClinical cloud platform. Save over 70% of the effort involved in toggling between numerous tools, coordination between multiple channels of engagement between study team and participants, participants use their own mobile device, and study specific data governance. ...
ByJeeva Informatics Solutions Inc. based in Manassas, VIRGINIA (USA)
CROs can differentiate their services to sponsors with the white-labeled Jeeva eClinical technology platform advantage. Maximize the probability of winning contracts with sponsors by differentiating your proposals with Jeeva eClinical cloud. Sponsors want to know how you can ensure faster patient recruitment and better retention compared to your peers. Jeeva can rapidly turnaround with a budget proposal and branded material for inclusion in your response to sponsor RFPs and support you throughout the ...
ByJeeva Informatics Solutions Inc. based in Manassas, VIRGINIA (USA)
Many genetic lung diseases have associated complications involving other organs that, if detected early, can be treated effectively. ...
ByTesis Biosciences based in Scottsdale, ARIZONA (USA)